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FDA Approvals of Brand-Name Prescription Drugs in 2014 I. New Pharmaceuticals: New Molecular Entities and New Biologic License Applications Approved in 2014 Akynzeo (NME; new combination) (Netupitant [NME] and palonosetron; Eisai) Class/route: Antiemetic; oral Indication: For the prevention of acute and delayed nausea and vomiting associated with initial and repeat courses of chemotherapy Beleodaq (NME) (Belinostat; Spectrum Pharmaceuticals) Class/route: Histone deacetylase inhibitor; intravenous Indication: For the treatment of patients with relapsed or refractory peripheral T-cell lymphoma, a type of NHL Approval considerations: Accelerated approval, priority review, orphan drug Belsomra (NME) (Suvorexant; Merck, Sharpe & Dohme Corporation) Class/route: Orexin receptor antagonist; oral Indication: For the treatment of insomnia characterized by difficulties with sleep onset and/or sleep maintenance Blincyto (BLA) (Blinatumomab; Amgen) Class/route: CD19-directed CD3 T-cell engager; intravenous Indication: Treatment of Philadelphia chromosome– negative relapsed or refractory B-cell precursor acute lymphoblastic leukemia Approval considerations: Breakthrough therapy, priority review, orphan drug, REMS
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Class/route: Human vascular endothelial growth factor receptor-2 antagonist; intravenous Indications: As a single agent or in combination with paclitaxel, for the treatment of advanced gastric or gastroesophageal junction adenocarcinoma, in patients with disease progression during or after previous fluoropyrimidine- or platinum-containing chemotherapy; in combination with docetaxel, for the treatment of metastatic non–small-cell lung cancer in patients with disease progression during or after platinum- based chemotherapy Approval considerations: Priority review, orphan drug Dalvance (NME) (Dalbavancin; Durata Therapeutics) Class/route: Glycopeptide antibiotic; intravenous Indication: Treatment of adults with acute bacterial skin and skin structure infections caused by susceptible isolates of the gram-positive microorganisms Staphylococcus aureus, Streptococcus pyogenes, Streptococcus agalactiae, and Streptococcus anginosus Approval consideration: Priority review, QIDP
Cerdelga (NME) (Eliglustat; Genzyme) Class/route: Glucosylceramide synthase inhibitor; oral Indication: For long-term treatment of adults with Gaucher disease type 1 who are CYP2D6 extensive metabolizers, intermediate metabolizers, or poor metabolizers as detected by an FDA-cleared test Approval considerations: Priority review, orphan drug
Entyvio (BLA) (Vedolizumab; Takeda Pharmaceuticals) Class/route: Integrin receptor antagonist; intravenous Indications: Treatment of adults with moderately to severely active ulcerative colitis who have had an inadequate response with, lost response to, or were intolerant to a TNF blocker or an immunomodulator; or had an inadequate response with, were intolerant to, or demonstrated dependence on corticosteroids; treatment of adults with moderately to severely active Crohn’s disease who have had an inadequate response with, lost response to, or were intolerant to a TNF blocker or immunomodulator; or had an inadequate response with, were intolerant to, or demonstrated dependence on corticosteroids Approval consideration: Priority review
Cyramza (BLA) (Ramucirumab; Eli Lilly)
Esbriet (NME) (Pirfenidone; InterMune)
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Class/route: Pyridone; oral Indication: Treatment of idiopathic pulmonary fibrosis Approval considerations: Breakthrough therapy, fast track, priority review, orphan drug Farxiga (NME) (Dapaglifozin; Bristol-Myers Squibb/AstraZeneca) Class/route: SGLT2 inhibitor; oral Indications: As an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes mellitus Harvoni (NME; new combination) (Ledipasvir [NME] and sofosbuvir; Gilead Sciences) Classes/route: Fixed-dose combination of an HCV NS5A inhibitor and an HCV nucleotide analog NS5B polymerase inhibitor; oral Indication: Treatment of genotype 1 chronic hepatitis C infection in adults; first fixed-dose combination without interferon or ribavirin Approval considerations: Priority review, breakthrough therapy [2015 new dosages approved: 5 mg; 10 mg] Hetlioz (NME) (Tasimelteon; Vanda Pharmaceuticals) Class/route: Melatonin receptor agonist; oral Indication: Treatment of non–24-hour sleep-wake disorder Approval considerations: Priority review, orphan drug Impavido (NME) (Miltefosine; Paladin Therapeutics) Class/route: Anthelmintic antineoplastic; oral Indications: For the treatment of individuals aged ≥12 years weighing ≥66 lb (30 kg) with visceral leishmaniasis resulting from Leishmania donovani; cutaneous leishmaniasis resulting from Leishmania braziliensis, Leishmania guyanensis, and Leishmania panamensis; and mucosal leishmaniasis resulting from L braziliensis Approval considerations: Fast track, priority review, orphan drug Jardiance (NME) (Empagliflozin; Boehringer Ingelheim) Class/route: SGLT2 inhibitor; oral Indication: As an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes mellitus Jublia (NME) (Efinaconazole; Valeant Pharmaceuticals International) Class/route: Triazole antifungal agent; topical Indication: For the treatment of onychomycosis of the toenails resulting from Trichophyton rubrum and Trichophyton mentagrophytes
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Kerydin (NME) (Tavaborole; Anacor Pharmaceuticals) Class/route: Oxaborole antifungal; topical Indication: For the treatment of onychomycosis of the toenails resulting from Trichophyton rubrum or Trichophyton mentagrophytes Keytruda (BLA) (Pembrolizumab; Merck) Class/route: PD-1–blocking antibody; intravenous Indication: Treatment of patients with unresectable or metastatic melanoma and disease progression after ipilimumab therapy and, if BRAF V600 mutation– positive, a BRAF inhibitor Approval considerations: Breakthrough therapy, accelerated approval, priority review, orphan drug Lumason (NME) (Sulfur hexafluoride lipid microsphere; Bracco Diagnostics) Class/route: Ultrasound contrast agent; intravenous Indication: For use in patients with suboptimal echocardiograms to opacify the left-ventricular chamber and to improve the delineation of the left-ventricular endocardial border Lynparza (NME) (Olaparib; AstraZeneca) Class/route: Poly (ADP-ribose) polymerase inhibitor; oral Indications: For advanced ovarian cancer in patients with deleterious or suspected deleterious germline BRCA mutation (as detected by an FDA-approved test) who have been treated with 3 or more previous lines of chemotherapy Approval considerations: Accelerated approval, priority review, orphan drug Movantik (NME) (Naloxegol; AstraZeneca) Class/route: Peripherally acting opioid receptor antagonists; oral Indication: For opioid-induced constipation in adults with chronic noncancer pain Approval consideration: Priority review Myalept (BLA) (Metreleptin; Amylin Pharmaceuticals) Class/route: Leptin analog; subcutaneous Indication: For the treatment of complications of leptin deficiency, as replacement therapy, in addition to diet, in patients with congenital generalized or acquired generalized lipodystrophy Approval considerations: Priority review, orphan drug, REMS
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Neuraceq (NME) (Florbetaben F18 injection; Piramal Imaging) Class/route: Radioactive diagnostic agent; intravenous Indication: For positron emission tomography imaging of the brain to estimate beta-amyloid neuritic plaque density in adults with cognitive impairment who are being evaluated for Alzheimer’s disease and other causes of cognitive decline Northera (NME) (Droxidopa; Chelsea Therapeutics) Class/route: Vasopressor; oral Indication: Treatment of orthostatic dizziness, lightheadedness, or the “feeling that you are about to black out” in adults with symptomatic neurogenic orthostatic hypotension caused by primary autonomic failure (Parkinson’s disease, multiple system atrophy, and pure autonomic failure), dopamine beta-hydroxylase deficiency, and nondiabetic autonomic neuropathy Approval considerations: Accelerated approval, priority review, orphan drug Ofev (NME) (Nintedanib; Boehringer Ingelheim) Class/route: Tyrosine kinase inhibitor; oral Indication: Treatment of idiopathic pulmonary fibrosis Approval considerations: Breakthrough therapy, fast track, priority review, orphan drug Opdivo (BLA) (Nivolumab; Bristol-Myers Squibb) Class/route: PD-1–blocking antibody; intravenous Indication: Treatment of unresectable or metastatic melanoma and disease progression after ipilimumab and, if BRAF V600 mutation–positive, a BRAF inhibitor Approval considerations: Breakthrough therapy, accelerated approval, priority review, orphan drug [2015 new indication: lung cancer] Orbactiv (NME) (Oritavancin; The Medicines Company) Class/route: Lipoglycopeptide antibacterial drug; intravenous Indication: Treatment of adults with acute bacterial skin and skin structure infections caused or suspected to be caused by susceptible bacteria, including S aureus (including methicillin-susceptible and methicillin- resistant strains), various Streptococcus species, and Enterococcus faecalis Approval considerations: Priority review, QIDP Otezla (NME) (Apremilast; Celgene)
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Class/route: Phosphodiesterase 4 inhibitor; oral Indications: Treatment of adults with active psoriatic arthritis, and patients with moderate-to-severe plaque psoriasis who are candidates for phototherapy or systemic therapy Plegridy (BLA) (Peginterferon beta-1a; Biogen Idec) Class/route: Interferon beta; subcutaneous Indication: Treatment of relapsing forms of multiple sclerosis Rapivab (NME) (Peramivir; BioCryst Pharmaceuticals) Class/route: Neuraminidase inhibitor; intravenous Indication: Treatment of acute uncomplicated influenza in patients aged ≥18 years who have been symptomatic for no more than 2 days Sivextro (NME) (Tedizolid phosphate; Cubist Pharmaceuticals) Class/route: Oxazolidinone-class antibacterial; intravenous and oral Indication: Treatment of adults with acute bacterial skin and skin structure infections caused by designated susceptible bacteria, including S aureus (including methicillin-resistant strains and methicillin-susceptible strains), various Streptococcus species, and E faecalis Approval considerations: Priority review, QIDP Striverdi Respimat (NME) (Olodaterol; Boehringer Ingelheim) Class/route: Long-acting beta2-adrenergic agonist; oral inhalation Indication: For long-term, once-daily maintenance bronchodilator treatment of airflow obstruction in patients with COPD, including chronic bronchitis and/or emphysema Sylvant (BLA) (Siltuximab; Janssen Biotech) Class/route: Interleukin-6 antagonist; intravenous Indication: Treatment of patients with multicentric Castleman’s disease who are HIV-negative and human herpesvirus-8–negative Approval considerations: Priority review, orphan drug Tanzeum (BLA) (Albiglutide; GlaxoSmithKline) Class/route: GLP-1 receptor agonist; subcutaneous Indication: As an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes mellitus Approval consideration: REMS
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Trulicity (BLA) (Dulaglutide; Lilly) Class/route: GLP-1 receptor agonist; subcutaneous Indication: As an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes mellitus Approval consideration: REMS Viekira Pak (NME; new combination) (Ombitasvir [NME], paritaprevir [NME], and ritonavir, copackaged with dasabuvir [NME]; AbbVie) Classes/route: HCV NS5A inhibitor, HCV NS3/4A protease inhibitor, and HCV nonnucleoside NS5B palm polymerase inhibitor; oral Indication: For the treatment, with or without ribavirin, of patients with genotype 1 chronic HCV infection, including those with compensated cirrhosis; fixed-dose combination Approval considerations: Breakthrough therapy, priority review Vimizim (BLA) (Elosulfase alfa; BioMarin Pharmaceutical) Class/route: Hydrolytic lysosomal glycosaminoglycan– specific enzyme; intravenous Indication: Treatment of mucopolysaccharidosis type IVA (Morquio A syndrome) Approval considerations: Priority review, orphan drug Xtoro (NME) (Finafloxacin; Alcon Laboratories) Class/route: Fluoroquinolone antimicrobial; topical otic suspension Indication: Treatment of acute otitis externa caused by susceptible strains of Pseudomonas aeruginosa and S aureus Approval consideration: Priority review
Zerbaxa (NME; new combination) (Ceftolozane [NME] and tazobactam; Cubist Pharmaceuticals) Class/route: A cephalosporin antibacterial drug and a beta-lactamase inhibitor; intravenous Indications: For the treatment of complicated intra-abdominal infections, in combination with metronidazole, and for the treatment of complicated urinary tract infections, including pyelonephritis Approval considerations: Priority review, QIDP Zontivity (NME) (Vorapaxar; Merck) Class/route: Protease-activated receptor-1 antagonist; oral Indication: For the reduction of thrombotic cardiovascular events in patients with a history of myocardial infarction or with peripheral arterial disease Zydelig (NME) (Idelalisib; Gilead Sciences) Class/route: Phosphoinositide-3 kinase-delta inhibitor; oral Indications: Treatment of relapsed chronic lymphocytic leukemia, in combination with rituximab, in patients for whom rituximab alone would be considered appropriate therapy because of other comorbidities; relapsed follicular B-cell NHL in patients who have received at least 2 previous systemic therapies; and relapsed small lymphocytic lymphoma in patients who have received at least 2 previous systemic therapies Approval considerations: Accelerated approval, priority review, orphan drug, REMS Zykadia (NME) (Ceritinib; Novartis) Class/route: ALK tyrosine kinase inhibitor; oral Indication: Treatment of ALK-positive metastatic non– small-cell lung cancer in patients who have progressed on or are intolerant to crizotinib Approval considerations: Breakthrough therapy, accelerated approval, priority review, orphan drug
ALK indicates anaplastic lymphoma kinase; BLA, biologic license application; COPD, chronic obstructive pulmonary disease; FDA, US Food and Drug Administration; GLP-1, glucagon-like peptide-1; HCV, hepatitis C virus; NHL, non-Hodgkin lymphoma; NME, new molecular entity; QIDP, Qualified Infectious Disease Product; PD-1, programmed-cell death 1; REMS, Risk Evaluation and Mitigation Strategy; SGLT2, sodium-glucose cotransporter 2; TNF, tumor necrosis factor.
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II. New Combinations and New Indications Approved in 2014 Afrezza (new indication) (Insulin human; MannKind Corporation) Class/route: Insulin human; inhaled powder New indication: Rapid-acting insulin to be used at the beginning of each meal to improve glycemic control in adults with type 1 or type 2 diabetes mellitus New formulation: Rapid-acting inhaled insulin Approval consideration: REMS Akynzeo (new combination) See NMEs/BLAs listing Arzerra (new indication) (Ofatumumab; GlaxoSmithKline) Class/route: CD20 monoclonal antibody; intravenous New indication: For first-line treatment, in combination with chlorambucil, of treatment-naïve patients with CLL for whom fludarabine-based therapy is inappropriate Existing indication: For the treatment of patients with CLL that is refractory to fludarabine or to alemtuzumab Avastin (new indications) (Bevacizumab; Genentech) Class/route: VEGF-specific angiogenesis inhibitor; intravenous New indications: For the treatment of persistent, recurrent, or metastatic cervical cancer in combination with paclitaxel and cisplatin or paclitaxel and topotecan; and for the treatment of platinum-resistant recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer, in combination with paclitaxel, pegylated liposomal doxorubicin, or topotecan Existing indications: First-line or second-line treatment of metastatic colorectal cancer with intravenous 5-fluorouracil–based chemotherapy; for second-line treatment of metastatic colorectal cancer in patients who have pro gressed on a first-line bevacizumab-containing regimen with fluoropyrimidine- irinotecan- or, fluoropyrimidine- oxaliplatin–based chemotherapy; with carboplatin and paclitaxel for first-line treatment of unresectable, locally advanced, recurrent or metastatic nonsquamous non– small-cell lung cancer; and as a single agent for adults with progressive glioblastoma after previous therapy; metastatic renal-cell carcinoma with interferon alfa Approval consideration: Priority review Contrave (new combination) (Naltrexone and bupropion; Takeda Pharmaceuticals) Classes/route: Opioid antagonist and aminoketone antidepressant; oral Indication: As an adjunct to a reduced-calorie diet and
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increased physical activity for chronic weight management in adults with an initial body mass index of ≥30 kg/m2 or ≥27 kg/m2 in the presence of at least ≥1 weight-related comorbidities Eliquis (new indications) (Apixaban; Bristol-Myers Squibb) Class/route: Factor Xa inhibitor anticoagulant; oral New indications: For prophylactic therapy of DVT that may lead to PE in patients who have undergone hip/ knee replacement surgery; for the treatment of DVT and PE, and for risk reduction of recurrent DVT and PE after initial therapy Existing indication: For risk reduction of stroke and systemic embolism in patients with nonvalvular atrial fibrillation Approval consideration: REMS Eylea (new indications) (Aflibercept; Regeneron Pharmaceuticals) Class/route: VEGF inhibitor; intravitreal New indications: For the treatment of diabetic macular edema; first VEGF inhibitor approved for dosing on a less than monthly basis for the treatment of diabetic macular edema Existing indications: For the treatment of neovascular (wet) age-related macular degeneration, and for the treatment of macular edema after retinal vein occlusion Approval considerations: Breakthrough therapy, priority review [2015 new indication: Diabetic retinopathy in patients with diabetic macular edema] Harvoni (new combination) See NMEs/BLAs listing Humira (new patient population) (Adalimumab; AbbVie) Class/route: Tumor necrosis factor blocker; subcutaneous New patient population: Pediatric Crohn’s disease patients aged ≥6 years Indications: For reducing the signs and symptoms, inducing major clinical response, inhibiting the progression of structural damage, and improving physical function in adults with moderately to severely active rheumatoid arthritis; reducing the signs and symptoms of moderately to severely active polyarticular juvenile idiopathic arthritis in patients aged ≥2 years; reducing the signs and symptoms, inhibiting the progression of structural damage, and improving physical function in adults with active psoriatic arthritis;
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reducing the signs and symptoms of active ankylosing spondylitis in adults; reducing the signs and symptoms and inducing and maintaining clinical remission in adults with moderately to severely active Crohn’s disease who have had an inadequate response to conventional therapy; reducing the signs and symptoms and inducing and maintaining clinical remission in patients aged ≥6 years with moderately to severely active Crohn’s disease who have had an inadequate response to corticosteroids or immunomodulators, such as azathioprine, 6-mercaptopurine, or methotrexate; inducing and sustaining clinical remission in adults with moderately to severely active ulcerative colitis who have had an inadequate response to immunosuppressants, such as corticosteroids, azathioprine, or 6-mercaptopurine; treatment of adults with moderate-to-severe chronic plaque psoriasis who are candidates for systemic therapy or phototherapy, and when other systemic therapies are medically less appropriate Imbruvica (new indication) (Ibrutinib; Pharmacyclics) Class/route: Bruton’s tyrosine kinase inhibitor; oral New indications: Treatment of patients with relapsed CLL who have received at least 1 previous therapy and treatment of patients with CLL with 17p deletion Existing indication: Treatment of patients with mantle- cell lymphoma who have received at least 1 previous therapy Approval considerations: Breakthrough therapy, priority review, orphan drug [2015 new indication: Waldenström’s macroglobulinemia] Invega Sustenna (new indication) (Paliperidone palmitate; Janssen Pharmaceuticals) Class/route: Atypical antipsychotic; oral New indication: For the once-monthly treatment of schizoaffective disorder, as monotherapy and as an adjunct to mood stabilizers and/or antidepressants (first FDA approval for this indication) Indication: Treatment of schizophrenia Invokamet (new combination) (Canagliflozin and metformin hydrochloride; Janssen Pharmaceuticals) Classes/route: SGLT2 inhibitor and biguanide; oral Indication: As an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes mellitus who are not adequately controlled with a regimen containing metformin or canagliflozin or in patients already receiving canagliflozin and metformin
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Jakafi (new indication) (Ruxolitinib; Incyte Corporation) Class/route: Janus-associated kinase inhibitor; oral New indication: For the treatment of patients with polycythemia vera who have had an inadequate response to or are intolerant of hydroxyurea Existing indication: For the treatment of intermediate- risk or high-risk myelofibrosis Approval considerations: Priority review, orphan drug Lemtrada (new indication) (Alemtuzumab; Genzyme Corporation) Class/route: CD52-directed cytolytic monoclonal antibody; intravenous New indication: For the treatment of patients with relapsing forms of multiple sclerosis Existing indication: For the treatment of B-cell CLL Mekinist plus Tafinlar (new combination) (Trametinib and dabrafenib; GlaxoSmithKline) Classes/route: BRAF kinase inhibitor and MEK kinase inhibitor; oral Indication: For the treatment of advanced melanoma that is unresectable or metastatic in patients with the BRAF V600E or BRAF V600K mutation Approval considerations: Accelerated approval, priority review Minivelle (new indication) (Estradiol; Noven Pharmaceuticals) Class/route: Estrogen; transdermal New indication: For the prevention of postmenopausal osteoporosis Existing indication: Treatment of moderate-to-severe vasomotor symptoms resulting from menopause Namzaric (new combination) (Memantine hydrochloride extended release and donepezil hydrochloride; Forest Laboratories) Classes/route: NMDA receptor antagonist and acetylcholinesterase inhibitor; oral Indication: For the treatment of moderate-to-severe dementia of the Alzheimer’s type in patients stabilized on memantine hydrochloride and donepezil hydrochloride Obredon (new combination) (Hydrocodone bitartrate and guaifenesin; Novotec Pharma) Classes/route: Opioid antitussive and expectorant; oral Indications: For the symptomatic relief of cough and to loosen mucus associated with the common cold
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Olysio (new combination) (Simeprevir; Janssen; for use with sofosbuvir [Sovaldi; Gilead]) Class/route: HCV NS3/4A protease inhibitor; oral New combination: For use in combination with sofosbuvir as an all-oral, interferon-free, ribavirin-free treatment for adults with genotype 1 chronic HCV infection Existing combination: For the treatment of genotype 1 chronic HCV infection in combination with pegylated interferon and ribavirin regimen Omidria (new combination) (Phenylephrine and ketorolac; Omeros Corporation) Classes/route: Alpha 1-adrenergic receptor agonist and nonselective cyclooxygenase inhibitor; intraocular Indications: For the maintenance of pupil size by preventing intraoperative miosis and for reducing postoperative pain Onexton (new combination) (Clindamycin/benzoyl peroxide; Valeant Pharma ceuticals International) Class/route: Lincosamide antibacterial; topical gel Indication: For the topical treatment of acne vulgaris in patients aged ≥12 years Ozurdex (new indication) (Dexamethasone; Allergan) Class/route: Corticosteroid; intravitreal injection New indication: For the treatment of diabetic macular edema Existing indications: Treatment of macular edema after branch retinal vein occlusion or central retinal vein occlusion, and for the treatment of noninfectious uveitis affecting the posterior segment of the eye Priftin (new indication) (Rifapentine; sanofi-aventis) Class/route: Rifamycin antimycobacterial; oral New indication: In combination with isoniazid, for the treatment of latent tuberculosis infection in patients aged ≥2 years who are at high risk for progression to tuberculosis disease Existing indication: For the treatment of active pulmonary tuberculosis in adults and children aged ≥12 years caused by Mycobacterium tuberculosis Promacta (new indication) (Eltrombopag; GlaxoSmithKline) Class/route: Thrombopoietin receptor agonist; oral New indication: For the treatment of patients with severe aplastic anemia who have had an insufficient response to immunosuppressive therapy
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Existing indications: For the treatment of thrombocytopenia in patients with chronic immune (idiopathic) thrombocytopenia who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy; thrombocytopenia in patients with chronic hepatitis C to allow the initiation and maintenance of interferon-based therapy Approval considerations: Breakthrough therapy, priority review Saxenda (new version of Victoza; new indication) (Liraglutide [rDNA origin] injection; Novo Nordisk) Class/route: GLP-1 receptor agonist; subcutaneous New indication: For chronic weight management as an adjunct to a reduced-calorie diet and increased physical activity in adults with an initial body mass index ≥30 kg/m2 or ≥27 kg/m2 and at least 1 weight-related comorbid condition; this is a new version of Victoza (liraglutide [rDNA origin]), which is indicated for adults with type 2 diabetes Approval consideration: REMS Somatuline Depot (new indication) (Lanreotide; Ipsen Pharmaceuticals) Class/route: Somatostatin analog; injection New indication: For the treatment of patients with unresectable, moderately or well-differentiated, locally advanced or metastatic gastroenteropancreatic neuroendocrine tumors to improve progression-free survival Existing indication: For the long-term treatment of acromegalic patients who have had an inadequate response to or cannot be treated with surgery and/or radiotherapy Soolantra (new indication; new formulation) (Ivermectin 1%; Galderma) Class/route: Antibiotic-free anti-inflammatory cream; topical New indication: For once-daily treatment of patients with inflammatory lesions, bumps, and pimples, of rosacea; this is the first time ever ivermectin cream is approved for use in humans; it was previously only available for use (in different form) in livestock New formulation: 1% cream for humans Trezix (new combination) (Acetaminophen, caffeine, and dihydrocodeine bitartrate; WraSer Pharmaceuticals) Classes/route: Nonopiate, nonsalicylate analgesic and antipyretic; central nervous system and cardiovascular stimulant; and semisynthetic narcotic analgesic; oral Indication: Relief of moderate-to-moderately severe pain
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Triumeq (new combination) (Abacavir, dolutegravir, and lamivudine; ViiV Healthcare) Classes/route: Integrase strand transfer inhibitor (dolu tegravir) and nucleoside analogue reverse transcriptase inhibitors (abacavir and lamivudine); oral Indication: For the treatment of patients with HIV-1 infection Velcade (new indications) (Bortezomib; Millennium Pharmaceuticals) Class/routes: Proteasome inhibitor; intravenous, subcutaneous New indications: Retreatment of adults with multiple myeloma that had previously responded to bortezomib and relapsed at least 6 months after completion of previous treatment with bortezomib; first-line treatment of newly diagnosed patients with mantle-cell lymphoma Existing indications: For the treatment of patients with multiple myeloma or with mantle-cell lymphoma Viekira Pak (new combination) See NMEs/BLAs listing Vitekta (new combination) (Elvitegravir; Gilead Sciences) Class/route: HIV-1 integrase strand transfer inhibitor; oral Indication: Used in combination with an HIV protease inhibitor coadministered with ritonavir and with other antiretroviral drug(s) for the treatment of HIV-1 infection in antiretroviral treatment–experienced adults Xgeva (new indication) (Denosumab; Amgen) Class/route: RANK ligand inhibitor; subcutaneous New indication: Treatment of hypercalcemia of malignancy refractory to bisphosphonate therapy Existing indications: Prevention of skeletal-related events
in patients with bone metastases from solid tumors, and for the treatment of adults and skeletally mature adolescents with giant-cell tumor of bone that is unresectable or where surgical resection is likely to result in severe morbidity Approval consideration: Orphan drug Xigduo XR (new combination) (Dapagliflozin and metformin hydrochloride extended release; AstraZeneca) Classes/route: SGLT2 inhibitor and biguanide; oral Indication: To improve glycemic control in adults with type 2 diabetes when treatment with dapagliflozin and with metformin is appropriate Xolair (new indication) (Omalizumab; Genentech) Class/route: Anti-immunoglobulin E antibody; subcutaneous New indication: First biologic and first medicine approved by the FDA for the treatment of chronic idiopathic urticaria in adults and adolescents aged ≥12 years who remain symptomatic despite H1 antihistamine treatment Existing indication: Treatment of moderate-to-severe persistent asthma in patients aged ≥12 years with a positive skin test or in vitro reactivity to a perennial aeroallergen and symptoms that are inadequately controlled with inhaled corticosteroids Zerbaxa (new combination) See NMEs/BLAs listing Zorvolex (new indication) (Diclofenac; Iroko Pharmaceuticals) Class/route: NSAID; oral New indication: For the management of osteoarthritis pain Existing indication: For the management of mild-to- moderate acute pain
BLA indicates biologic license application; CLL, chronic lymphocytic leukemia; DVT, deep-vein thrombosis; GLP1, glucagon-like peptide-1; HCV, hepatitis C virus; NMDA, N-methyl-D-aspartate; NME, new molecular entity; NSAID, nonsteroidal anti-inflammatory drug; PE, pulmonary embolism; REMS, Risk Evaluation and Mitigation Strategy; SGLT2, sodium-glucose cotransporter 2; VEGF, vascular endothelial growth factor.
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III. New Formulations/Dosage Forms/Dosages/Patient Population Approved in 2014 Abilify Maintena (new dosage form) (Aripiprazole; Otsuka Pharmaceutical) Class/route: Atypical antipsychotic; intramuscular New dosage form: Extended-release injectable suspension in a prefilled dual-chamber syringe Indication: For the treatment of schizophrenia
Bunavail (new formulation) (Buprenorphine and naloxone; BioDelivery Sciences) Class/route: Partial opioid agonist; oral New formulation: Buccal film Indication: For the maintenance treatment of opioid dependence
Acticlate (new dosages) (Doxycycline hyclate USP; Aqua Pharmaceuticals) Class/route: Tetracycline-class antimicrobial; oral New dosages: 75-mg and 150-mg tablets Indications: For the treatment of Rickettsial infections, sexually transmitted infections, respiratory tract infections, specific bacterial infections, ophthalmic infections, and anthrax, including inhalational anthrax (postexposure); alternative treatment for selected infections when penicillin is contraindicated; adjunctive therapy in acute intestinal amebiasis and severe acne; prophylaxis of malaria
Bydureon (new dosage form) (Exenatide; AstraZeneca) Class/route: GLP-1 receptor agonist; subcutaneous New dosage form: Extended-release formula in a 2-mg pen; already available as a single-dose tray containing a 2-mg vial Indication: As an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes mellitus
Arnuity Ellipta (new formulation) (Fluticasone furoate; GlaxoSmithKline) Class/route: Corticosteroid; oral inhalation New formulation: Once-daily oral inhalation of 100 mcg or 200 mcg using Ellipta inhaler Indication: For the once-daily maintenance treatment of asthma as prophylactic therapy in patients aged ≥12 years Auryxia (new formulation) (Ferric citrate: Keryx Biopharmaceuticals) Class/route: Phosphate binder; oral New formulation: The first and only absorbable ironbased phosphate binder proven to manage hyperphosphatemia Indication: For the control of serum phosphorus levels in patients with chronic kidney disease on dialysis Aveed (new formulation) (Testosterone undecanoate; Endo Pharmaceuticals) Class/route: Androgen; intramuscular injection New formulation: Intramuscular; already available in oral, subcutaneous, buccal, and transdermal formulations Indication: For testosterone replacement therapy in adult men for conditions associated with a deficiency or absence of endogenous testosterone, such as congenital or acquired primary hypogonadism, or congenital or acquired hypogonadotropic hypogonadism Approval consideration: REMS
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Copaxone (new dosage) (Glatiramer acetate; Teva Pharmaceutical Industries) Class/route: Immunomodulator; subcutaneous injection New dosage: Three-times weekly of 40 mg/mL, a less frequent dose than the 20 mg/mL daily that is still available for use Indication: For the treatment of patients with relapsing form of multiple sclerosis Dyloject (new dosage form) (Diclofenac; Hospira) Class/route: NSAID; intravenous New dosage form: Intravenous bolus injection over 15 seconds Indications: For use in adults for the management of mild-to-moderate pain and for the management of moderate-to-severe pain alone or in combination with opioid analgesics Embeda (new formulation) (Morphine sulfate and naltrexone hydrochloride; Pfizer) Classes/route: Opioid agonist and opioid antagonist; oral New formulation: Has properties that are expected to reduce oral abuse when the drug is crushed; capsules should be swallowed whole to avoid exposure to a potentially fatal dose of morphine Indication: For the management of pain severe enough to require daily, around-the-clock, long-term opioid treatment and for which alternative treatment options are inadequate
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Epanova (new formulation) (Omega-3-carboxylic acids; AstraZeneca) Class/route: Omega-3 fatty acid; oral New formulation: Omega-3 in free fatty acid form Indications: As an adjunct to diet to reduce triglyceride levels in adults with severe (≥500 mg/dL) hypertriglyceridemia Erwinaze (new dosage form) (Asparaginase Erwinia chrysanthemi; Jazz Pharmaceuticals) Class/routes: Antineoplastic; intramuscular, intravenous New dosage form: Intravenous Indication: As a component of a multiagent chemotherapeutic regimen for the treatment of patients with acute lymphoblastic leukemia who have developed hypersensitivity to Escherichia coli–derived asparaginase Evzio (new formulation) (Naloxone hydrochloride; Kaleo) Class/routes: Opioid antagonist; intramuscular, subcutaneous New formulation: Handheld autoinjector Indication: For the treatment of proliferating infantile hemangioma requiring systemic therapy Approval consideration: Priority review
Hemangeol (new formulation) (Propranolol hydrochloride; Pierre Fabre Dermatologie) Class/route: Beta-adrenergic blocker; oral New formulation: Oral solution for pediatric use Indication: For the treatment of proliferating infantile hemangioma requiring systemic therapy Approval consideration: Orphan drug Hysingla ER (new formulation) (Hydrocodone bitartrate; Purdue Pharma) Class/route: Opioid agonist; oral New formulation: Extended-release opioid tablet with abuse-deterrent properties that make it difficult to crush, break, or dissolve, which is intended to reduce, but not entirely prevent, abuse of the drug when taken
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Iluvien (new dosage form) (Fluocinolone acetonide intravitreal implant; Alimera Sciences) Class/route: Corticosteroid; intravitreal injection New dosage form: Single injection of the microinsert provides sustained treatment for 36 months Indication: Treatment of diabetic macular edema in patients who have been previously treated with a course of corticosteroids and did not have a clinically significant rise in intraocular pressure Incruse Ellipta (new formulation) (Umeclidinium; GlaxoSmithKline) Class/route: Anticholinergic; oral inhalation New formulation: Once-daily bronchodilator Indication: For long-term, once-daily maintenance treatment of airflow obstruction in patients with COPD
Granix (new dosage form) (Tbo-filgrastim; Teva Pharmaceutical Industries) Class/route: Leukocyte growth factor; subcutaneous New dosage form: Injection for self-administration by patients and caregivers; previously only for administration by a healthcare professional Indication: For reduction in the duration of severe neutropenia in patients with nonmyeloid malignancies receiving myelosuppressive anticancer drugs associated with a clinically significant incidence of febrile neutropenia
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orally/chewed Indication: For the management of pain severe enough to require daily, around-the-clock, long-term opioid treatment and for which alternative treatment options are inadequate Approval considerations: Priority review, REMS
Izba (new dosage) (Travoprost; Alcon Laboratories) Class/route: Prostaglandin analog; ophthalmic solution New dosage: 0.003% ophthalmic solution; already available as a 0.004% solution Indication: For reduction of elevated intraocular pressure in patients with open-angle glaucoma or ocular hypertension Kalbitor (new patient population) (Ecallantide; Dyax Corporation) Class/route: Plasma kallikrein inhibitor; subcutaneous New patient population: For the treatment of patients aged ≥12 years; previously indicated only for adults Indication: Treatment of acute attacks of hereditary angioedema in patients aged ≥12 years Kalydeco (new patient population) (Ivacaftor; Vertex Pharmaceuticals Incorporated) Class/route: CFTR; oral New patient population: Treatment of cystic fibrosis in patients aged ≥6 years who have the R117H mutation in the CFTR gene Indication: For the treatment of cystic fibrosis in patients aged ≥6 years who have 1 of the following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R [2015 new patient population: All indications were expanded to include patients aged 2-5 years]
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Kerydin (new dosage form) (Tavaborole; Anacor Pharmaceuticals) Class/route: Oxaborole antifungal; topical New dosage form: First oxaborole antifungal available as a topical solution Indication: Topical treatment of onychomycosis of the toenails from Trichophyton rubrum or Trichophyton mentagrophytes Lumizyme (new patient population) (Alglucosidase alfa; Genzyme Corporation) Class/route: Hydrolytic lysosomal glycogen-specific enzyme; intravenous New patient population: Treatment of patients with infantile-onset Pompe disease, including patients aged
FDA Approvals of Brand-Name Prescription Drugs in 2014 I. New Pharmaceuticals: New Molecular Entities and New Biologic License Applications Approved in 2014 Akynzeo (NME; new combination) (Netupitant [NME] and palonosetron; Eisai) Class/route: Antiemetic; oral Indication: For the prevention of acute and delayed nausea and vomiting associated with initial and repeat courses of chemotherapy Beleodaq (NME) (Belinostat; Spectrum Pharmaceuticals) Class/route: Histone deacetylase inhibitor; intravenous Indication: For the treatment of patients with relapsed or refractory peripheral T-cell lymphoma, a type of NHL Approval considerations: Accelerated approval, priority review, orphan drug Belsomra (NME) (Suvorexant; Merck, Sharpe & Dohme Corporation) Class/route: Orexin receptor antagonist; oral Indication: For the treatment of insomnia characterized by difficulties with sleep onset and/or sleep maintenance Blincyto (BLA) (Blinatumomab; Amgen) Class/route: CD19-directed CD3 T-cell engager; intravenous Indication: Treatment of Philadelphia chromosome– negative relapsed or refractory B-cell precursor acute lymphoblastic leukemia Approval considerations: Breakthrough therapy, priority review, orphan drug, REMS
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Class/route: Human vascular endothelial growth factor receptor-2 antagonist; intravenous Indications: As a single agent or in combination with paclitaxel, for the treatment of advanced gastric or gastroesophageal junction adenocarcinoma, in patients with disease progression during or after previous fluoropyrimidine- or platinum-containing chemotherapy; in combination with docetaxel, for the treatment of metastatic non–small-cell lung cancer in patients with disease progression during or after platinum- based chemotherapy Approval considerations: Priority review, orphan drug Dalvance (NME) (Dalbavancin; Durata Therapeutics) Class/route: Glycopeptide antibiotic; intravenous Indication: Treatment of adults with acute bacterial skin and skin structure infections caused by susceptible isolates of the gram-positive microorganisms Staphylococcus aureus, Streptococcus pyogenes, Streptococcus agalactiae, and Streptococcus anginosus Approval consideration: Priority review, QIDP
Cerdelga (NME) (Eliglustat; Genzyme) Class/route: Glucosylceramide synthase inhibitor; oral Indication: For long-term treatment of adults with Gaucher disease type 1 who are CYP2D6 extensive metabolizers, intermediate metabolizers, or poor metabolizers as detected by an FDA-cleared test Approval considerations: Priority review, orphan drug
Entyvio (BLA) (Vedolizumab; Takeda Pharmaceuticals) Class/route: Integrin receptor antagonist; intravenous Indications: Treatment of adults with moderately to severely active ulcerative colitis who have had an inadequate response with, lost response to, or were intolerant to a TNF blocker or an immunomodulator; or had an inadequate response with, were intolerant to, or demonstrated dependence on corticosteroids; treatment of adults with moderately to severely active Crohn’s disease who have had an inadequate response with, lost response to, or were intolerant to a TNF blocker or immunomodulator; or had an inadequate response with, were intolerant to, or demonstrated dependence on corticosteroids Approval consideration: Priority review
Cyramza (BLA) (Ramucirumab; Eli Lilly)
Esbriet (NME) (Pirfenidone; InterMune)
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Class/route: Pyridone; oral Indication: Treatment of idiopathic pulmonary fibrosis Approval considerations: Breakthrough therapy, fast track, priority review, orphan drug Farxiga (NME) (Dapaglifozin; Bristol-Myers Squibb/AstraZeneca) Class/route: SGLT2 inhibitor; oral Indications: As an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes mellitus Harvoni (NME; new combination) (Ledipasvir [NME] and sofosbuvir; Gilead Sciences) Classes/route: Fixed-dose combination of an HCV NS5A inhibitor and an HCV nucleotide analog NS5B polymerase inhibitor; oral Indication: Treatment of genotype 1 chronic hepatitis C infection in adults; first fixed-dose combination without interferon or ribavirin Approval considerations: Priority review, breakthrough therapy [2015 new dosages approved: 5 mg; 10 mg] Hetlioz (NME) (Tasimelteon; Vanda Pharmaceuticals) Class/route: Melatonin receptor agonist; oral Indication: Treatment of non–24-hour sleep-wake disorder Approval considerations: Priority review, orphan drug Impavido (NME) (Miltefosine; Paladin Therapeutics) Class/route: Anthelmintic antineoplastic; oral Indications: For the treatment of individuals aged ≥12 years weighing ≥66 lb (30 kg) with visceral leishmaniasis resulting from Leishmania donovani; cutaneous leishmaniasis resulting from Leishmania braziliensis, Leishmania guyanensis, and Leishmania panamensis; and mucosal leishmaniasis resulting from L braziliensis Approval considerations: Fast track, priority review, orphan drug Jardiance (NME) (Empagliflozin; Boehringer Ingelheim) Class/route: SGLT2 inhibitor; oral Indication: As an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes mellitus Jublia (NME) (Efinaconazole; Valeant Pharmaceuticals International) Class/route: Triazole antifungal agent; topical Indication: For the treatment of onychomycosis of the toenails resulting from Trichophyton rubrum and Trichophyton mentagrophytes
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Kerydin (NME) (Tavaborole; Anacor Pharmaceuticals) Class/route: Oxaborole antifungal; topical Indication: For the treatment of onychomycosis of the toenails resulting from Trichophyton rubrum or Trichophyton mentagrophytes Keytruda (BLA) (Pembrolizumab; Merck) Class/route: PD-1–blocking antibody; intravenous Indication: Treatment of patients with unresectable or metastatic melanoma and disease progression after ipilimumab therapy and, if BRAF V600 mutation– positive, a BRAF inhibitor Approval considerations: Breakthrough therapy, accelerated approval, priority review, orphan drug Lumason (NME) (Sulfur hexafluoride lipid microsphere; Bracco Diagnostics) Class/route: Ultrasound contrast agent; intravenous Indication: For use in patients with suboptimal echocardiograms to opacify the left-ventricular chamber and to improve the delineation of the left-ventricular endocardial border Lynparza (NME) (Olaparib; AstraZeneca) Class/route: Poly (ADP-ribose) polymerase inhibitor; oral Indications: For advanced ovarian cancer in patients with deleterious or suspected deleterious germline BRCA mutation (as detected by an FDA-approved test) who have been treated with 3 or more previous lines of chemotherapy Approval considerations: Accelerated approval, priority review, orphan drug Movantik (NME) (Naloxegol; AstraZeneca) Class/route: Peripherally acting opioid receptor antagonists; oral Indication: For opioid-induced constipation in adults with chronic noncancer pain Approval consideration: Priority review Myalept (BLA) (Metreleptin; Amylin Pharmaceuticals) Class/route: Leptin analog; subcutaneous Indication: For the treatment of complications of leptin deficiency, as replacement therapy, in addition to diet, in patients with congenital generalized or acquired generalized lipodystrophy Approval considerations: Priority review, orphan drug, REMS
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Neuraceq (NME) (Florbetaben F18 injection; Piramal Imaging) Class/route: Radioactive diagnostic agent; intravenous Indication: For positron emission tomography imaging of the brain to estimate beta-amyloid neuritic plaque density in adults with cognitive impairment who are being evaluated for Alzheimer’s disease and other causes of cognitive decline Northera (NME) (Droxidopa; Chelsea Therapeutics) Class/route: Vasopressor; oral Indication: Treatment of orthostatic dizziness, lightheadedness, or the “feeling that you are about to black out” in adults with symptomatic neurogenic orthostatic hypotension caused by primary autonomic failure (Parkinson’s disease, multiple system atrophy, and pure autonomic failure), dopamine beta-hydroxylase deficiency, and nondiabetic autonomic neuropathy Approval considerations: Accelerated approval, priority review, orphan drug Ofev (NME) (Nintedanib; Boehringer Ingelheim) Class/route: Tyrosine kinase inhibitor; oral Indication: Treatment of idiopathic pulmonary fibrosis Approval considerations: Breakthrough therapy, fast track, priority review, orphan drug Opdivo (BLA) (Nivolumab; Bristol-Myers Squibb) Class/route: PD-1–blocking antibody; intravenous Indication: Treatment of unresectable or metastatic melanoma and disease progression after ipilimumab and, if BRAF V600 mutation–positive, a BRAF inhibitor Approval considerations: Breakthrough therapy, accelerated approval, priority review, orphan drug [2015 new indication: lung cancer] Orbactiv (NME) (Oritavancin; The Medicines Company) Class/route: Lipoglycopeptide antibacterial drug; intravenous Indication: Treatment of adults with acute bacterial skin and skin structure infections caused or suspected to be caused by susceptible bacteria, including S aureus (including methicillin-susceptible and methicillin- resistant strains), various Streptococcus species, and Enterococcus faecalis Approval considerations: Priority review, QIDP Otezla (NME) (Apremilast; Celgene)
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Class/route: Phosphodiesterase 4 inhibitor; oral Indications: Treatment of adults with active psoriatic arthritis, and patients with moderate-to-severe plaque psoriasis who are candidates for phototherapy or systemic therapy Plegridy (BLA) (Peginterferon beta-1a; Biogen Idec) Class/route: Interferon beta; subcutaneous Indication: Treatment of relapsing forms of multiple sclerosis Rapivab (NME) (Peramivir; BioCryst Pharmaceuticals) Class/route: Neuraminidase inhibitor; intravenous Indication: Treatment of acute uncomplicated influenza in patients aged ≥18 years who have been symptomatic for no more than 2 days Sivextro (NME) (Tedizolid phosphate; Cubist Pharmaceuticals) Class/route: Oxazolidinone-class antibacterial; intravenous and oral Indication: Treatment of adults with acute bacterial skin and skin structure infections caused by designated susceptible bacteria, including S aureus (including methicillin-resistant strains and methicillin-susceptible strains), various Streptococcus species, and E faecalis Approval considerations: Priority review, QIDP Striverdi Respimat (NME) (Olodaterol; Boehringer Ingelheim) Class/route: Long-acting beta2-adrenergic agonist; oral inhalation Indication: For long-term, once-daily maintenance bronchodilator treatment of airflow obstruction in patients with COPD, including chronic bronchitis and/or emphysema Sylvant (BLA) (Siltuximab; Janssen Biotech) Class/route: Interleukin-6 antagonist; intravenous Indication: Treatment of patients with multicentric Castleman’s disease who are HIV-negative and human herpesvirus-8–negative Approval considerations: Priority review, orphan drug Tanzeum (BLA) (Albiglutide; GlaxoSmithKline) Class/route: GLP-1 receptor agonist; subcutaneous Indication: As an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes mellitus Approval consideration: REMS
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Trulicity (BLA) (Dulaglutide; Lilly) Class/route: GLP-1 receptor agonist; subcutaneous Indication: As an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes mellitus Approval consideration: REMS Viekira Pak (NME; new combination) (Ombitasvir [NME], paritaprevir [NME], and ritonavir, copackaged with dasabuvir [NME]; AbbVie) Classes/route: HCV NS5A inhibitor, HCV NS3/4A protease inhibitor, and HCV nonnucleoside NS5B palm polymerase inhibitor; oral Indication: For the treatment, with or without ribavirin, of patients with genotype 1 chronic HCV infection, including those with compensated cirrhosis; fixed-dose combination Approval considerations: Breakthrough therapy, priority review Vimizim (BLA) (Elosulfase alfa; BioMarin Pharmaceutical) Class/route: Hydrolytic lysosomal glycosaminoglycan– specific enzyme; intravenous Indication: Treatment of mucopolysaccharidosis type IVA (Morquio A syndrome) Approval considerations: Priority review, orphan drug Xtoro (NME) (Finafloxacin; Alcon Laboratories) Class/route: Fluoroquinolone antimicrobial; topical otic suspension Indication: Treatment of acute otitis externa caused by susceptible strains of Pseudomonas aeruginosa and S aureus Approval consideration: Priority review
Zerbaxa (NME; new combination) (Ceftolozane [NME] and tazobactam; Cubist Pharmaceuticals) Class/route: A cephalosporin antibacterial drug and a beta-lactamase inhibitor; intravenous Indications: For the treatment of complicated intra-abdominal infections, in combination with metronidazole, and for the treatment of complicated urinary tract infections, including pyelonephritis Approval considerations: Priority review, QIDP Zontivity (NME) (Vorapaxar; Merck) Class/route: Protease-activated receptor-1 antagonist; oral Indication: For the reduction of thrombotic cardiovascular events in patients with a history of myocardial infarction or with peripheral arterial disease Zydelig (NME) (Idelalisib; Gilead Sciences) Class/route: Phosphoinositide-3 kinase-delta inhibitor; oral Indications: Treatment of relapsed chronic lymphocytic leukemia, in combination with rituximab, in patients for whom rituximab alone would be considered appropriate therapy because of other comorbidities; relapsed follicular B-cell NHL in patients who have received at least 2 previous systemic therapies; and relapsed small lymphocytic lymphoma in patients who have received at least 2 previous systemic therapies Approval considerations: Accelerated approval, priority review, orphan drug, REMS Zykadia (NME) (Ceritinib; Novartis) Class/route: ALK tyrosine kinase inhibitor; oral Indication: Treatment of ALK-positive metastatic non– small-cell lung cancer in patients who have progressed on or are intolerant to crizotinib Approval considerations: Breakthrough therapy, accelerated approval, priority review, orphan drug
ALK indicates anaplastic lymphoma kinase; BLA, biologic license application; COPD, chronic obstructive pulmonary disease; FDA, US Food and Drug Administration; GLP-1, glucagon-like peptide-1; HCV, hepatitis C virus; NHL, non-Hodgkin lymphoma; NME, new molecular entity; QIDP, Qualified Infectious Disease Product; PD-1, programmed-cell death 1; REMS, Risk Evaluation and Mitigation Strategy; SGLT2, sodium-glucose cotransporter 2; TNF, tumor necrosis factor.
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II. New Combinations and New Indications Approved in 2014 Afrezza (new indication) (Insulin human; MannKind Corporation) Class/route: Insulin human; inhaled powder New indication: Rapid-acting insulin to be used at the beginning of each meal to improve glycemic control in adults with type 1 or type 2 diabetes mellitus New formulation: Rapid-acting inhaled insulin Approval consideration: REMS Akynzeo (new combination) See NMEs/BLAs listing Arzerra (new indication) (Ofatumumab; GlaxoSmithKline) Class/route: CD20 monoclonal antibody; intravenous New indication: For first-line treatment, in combination with chlorambucil, of treatment-naïve patients with CLL for whom fludarabine-based therapy is inappropriate Existing indication: For the treatment of patients with CLL that is refractory to fludarabine or to alemtuzumab Avastin (new indications) (Bevacizumab; Genentech) Class/route: VEGF-specific angiogenesis inhibitor; intravenous New indications: For the treatment of persistent, recurrent, or metastatic cervical cancer in combination with paclitaxel and cisplatin or paclitaxel and topotecan; and for the treatment of platinum-resistant recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer, in combination with paclitaxel, pegylated liposomal doxorubicin, or topotecan Existing indications: First-line or second-line treatment of metastatic colorectal cancer with intravenous 5-fluorouracil–based chemotherapy; for second-line treatment of metastatic colorectal cancer in patients who have pro gressed on a first-line bevacizumab-containing regimen with fluoropyrimidine- irinotecan- or, fluoropyrimidine- oxaliplatin–based chemotherapy; with carboplatin and paclitaxel for first-line treatment of unresectable, locally advanced, recurrent or metastatic nonsquamous non– small-cell lung cancer; and as a single agent for adults with progressive glioblastoma after previous therapy; metastatic renal-cell carcinoma with interferon alfa Approval consideration: Priority review Contrave (new combination) (Naltrexone and bupropion; Takeda Pharmaceuticals) Classes/route: Opioid antagonist and aminoketone antidepressant; oral Indication: As an adjunct to a reduced-calorie diet and
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increased physical activity for chronic weight management in adults with an initial body mass index of ≥30 kg/m2 or ≥27 kg/m2 in the presence of at least ≥1 weight-related comorbidities Eliquis (new indications) (Apixaban; Bristol-Myers Squibb) Class/route: Factor Xa inhibitor anticoagulant; oral New indications: For prophylactic therapy of DVT that may lead to PE in patients who have undergone hip/ knee replacement surgery; for the treatment of DVT and PE, and for risk reduction of recurrent DVT and PE after initial therapy Existing indication: For risk reduction of stroke and systemic embolism in patients with nonvalvular atrial fibrillation Approval consideration: REMS Eylea (new indications) (Aflibercept; Regeneron Pharmaceuticals) Class/route: VEGF inhibitor; intravitreal New indications: For the treatment of diabetic macular edema; first VEGF inhibitor approved for dosing on a less than monthly basis for the treatment of diabetic macular edema Existing indications: For the treatment of neovascular (wet) age-related macular degeneration, and for the treatment of macular edema after retinal vein occlusion Approval considerations: Breakthrough therapy, priority review [2015 new indication: Diabetic retinopathy in patients with diabetic macular edema] Harvoni (new combination) See NMEs/BLAs listing Humira (new patient population) (Adalimumab; AbbVie) Class/route: Tumor necrosis factor blocker; subcutaneous New patient population: Pediatric Crohn’s disease patients aged ≥6 years Indications: For reducing the signs and symptoms, inducing major clinical response, inhibiting the progression of structural damage, and improving physical function in adults with moderately to severely active rheumatoid arthritis; reducing the signs and symptoms of moderately to severely active polyarticular juvenile idiopathic arthritis in patients aged ≥2 years; reducing the signs and symptoms, inhibiting the progression of structural damage, and improving physical function in adults with active psoriatic arthritis;
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reducing the signs and symptoms of active ankylosing spondylitis in adults; reducing the signs and symptoms and inducing and maintaining clinical remission in adults with moderately to severely active Crohn’s disease who have had an inadequate response to conventional therapy; reducing the signs and symptoms and inducing and maintaining clinical remission in patients aged ≥6 years with moderately to severely active Crohn’s disease who have had an inadequate response to corticosteroids or immunomodulators, such as azathioprine, 6-mercaptopurine, or methotrexate; inducing and sustaining clinical remission in adults with moderately to severely active ulcerative colitis who have had an inadequate response to immunosuppressants, such as corticosteroids, azathioprine, or 6-mercaptopurine; treatment of adults with moderate-to-severe chronic plaque psoriasis who are candidates for systemic therapy or phototherapy, and when other systemic therapies are medically less appropriate Imbruvica (new indication) (Ibrutinib; Pharmacyclics) Class/route: Bruton’s tyrosine kinase inhibitor; oral New indications: Treatment of patients with relapsed CLL who have received at least 1 previous therapy and treatment of patients with CLL with 17p deletion Existing indication: Treatment of patients with mantle- cell lymphoma who have received at least 1 previous therapy Approval considerations: Breakthrough therapy, priority review, orphan drug [2015 new indication: Waldenström’s macroglobulinemia] Invega Sustenna (new indication) (Paliperidone palmitate; Janssen Pharmaceuticals) Class/route: Atypical antipsychotic; oral New indication: For the once-monthly treatment of schizoaffective disorder, as monotherapy and as an adjunct to mood stabilizers and/or antidepressants (first FDA approval for this indication) Indication: Treatment of schizophrenia Invokamet (new combination) (Canagliflozin and metformin hydrochloride; Janssen Pharmaceuticals) Classes/route: SGLT2 inhibitor and biguanide; oral Indication: As an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes mellitus who are not adequately controlled with a regimen containing metformin or canagliflozin or in patients already receiving canagliflozin and metformin
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Jakafi (new indication) (Ruxolitinib; Incyte Corporation) Class/route: Janus-associated kinase inhibitor; oral New indication: For the treatment of patients with polycythemia vera who have had an inadequate response to or are intolerant of hydroxyurea Existing indication: For the treatment of intermediate- risk or high-risk myelofibrosis Approval considerations: Priority review, orphan drug Lemtrada (new indication) (Alemtuzumab; Genzyme Corporation) Class/route: CD52-directed cytolytic monoclonal antibody; intravenous New indication: For the treatment of patients with relapsing forms of multiple sclerosis Existing indication: For the treatment of B-cell CLL Mekinist plus Tafinlar (new combination) (Trametinib and dabrafenib; GlaxoSmithKline) Classes/route: BRAF kinase inhibitor and MEK kinase inhibitor; oral Indication: For the treatment of advanced melanoma that is unresectable or metastatic in patients with the BRAF V600E or BRAF V600K mutation Approval considerations: Accelerated approval, priority review Minivelle (new indication) (Estradiol; Noven Pharmaceuticals) Class/route: Estrogen; transdermal New indication: For the prevention of postmenopausal osteoporosis Existing indication: Treatment of moderate-to-severe vasomotor symptoms resulting from menopause Namzaric (new combination) (Memantine hydrochloride extended release and donepezil hydrochloride; Forest Laboratories) Classes/route: NMDA receptor antagonist and acetylcholinesterase inhibitor; oral Indication: For the treatment of moderate-to-severe dementia of the Alzheimer’s type in patients stabilized on memantine hydrochloride and donepezil hydrochloride Obredon (new combination) (Hydrocodone bitartrate and guaifenesin; Novotec Pharma) Classes/route: Opioid antitussive and expectorant; oral Indications: For the symptomatic relief of cough and to loosen mucus associated with the common cold
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Olysio (new combination) (Simeprevir; Janssen; for use with sofosbuvir [Sovaldi; Gilead]) Class/route: HCV NS3/4A protease inhibitor; oral New combination: For use in combination with sofosbuvir as an all-oral, interferon-free, ribavirin-free treatment for adults with genotype 1 chronic HCV infection Existing combination: For the treatment of genotype 1 chronic HCV infection in combination with pegylated interferon and ribavirin regimen Omidria (new combination) (Phenylephrine and ketorolac; Omeros Corporation) Classes/route: Alpha 1-adrenergic receptor agonist and nonselective cyclooxygenase inhibitor; intraocular Indications: For the maintenance of pupil size by preventing intraoperative miosis and for reducing postoperative pain Onexton (new combination) (Clindamycin/benzoyl peroxide; Valeant Pharma ceuticals International) Class/route: Lincosamide antibacterial; topical gel Indication: For the topical treatment of acne vulgaris in patients aged ≥12 years Ozurdex (new indication) (Dexamethasone; Allergan) Class/route: Corticosteroid; intravitreal injection New indication: For the treatment of diabetic macular edema Existing indications: Treatment of macular edema after branch retinal vein occlusion or central retinal vein occlusion, and for the treatment of noninfectious uveitis affecting the posterior segment of the eye Priftin (new indication) (Rifapentine; sanofi-aventis) Class/route: Rifamycin antimycobacterial; oral New indication: In combination with isoniazid, for the treatment of latent tuberculosis infection in patients aged ≥2 years who are at high risk for progression to tuberculosis disease Existing indication: For the treatment of active pulmonary tuberculosis in adults and children aged ≥12 years caused by Mycobacterium tuberculosis Promacta (new indication) (Eltrombopag; GlaxoSmithKline) Class/route: Thrombopoietin receptor agonist; oral New indication: For the treatment of patients with severe aplastic anemia who have had an insufficient response to immunosuppressive therapy
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Existing indications: For the treatment of thrombocytopenia in patients with chronic immune (idiopathic) thrombocytopenia who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy; thrombocytopenia in patients with chronic hepatitis C to allow the initiation and maintenance of interferon-based therapy Approval considerations: Breakthrough therapy, priority review Saxenda (new version of Victoza; new indication) (Liraglutide [rDNA origin] injection; Novo Nordisk) Class/route: GLP-1 receptor agonist; subcutaneous New indication: For chronic weight management as an adjunct to a reduced-calorie diet and increased physical activity in adults with an initial body mass index ≥30 kg/m2 or ≥27 kg/m2 and at least 1 weight-related comorbid condition; this is a new version of Victoza (liraglutide [rDNA origin]), which is indicated for adults with type 2 diabetes Approval consideration: REMS Somatuline Depot (new indication) (Lanreotide; Ipsen Pharmaceuticals) Class/route: Somatostatin analog; injection New indication: For the treatment of patients with unresectable, moderately or well-differentiated, locally advanced or metastatic gastroenteropancreatic neuroendocrine tumors to improve progression-free survival Existing indication: For the long-term treatment of acromegalic patients who have had an inadequate response to or cannot be treated with surgery and/or radiotherapy Soolantra (new indication; new formulation) (Ivermectin 1%; Galderma) Class/route: Antibiotic-free anti-inflammatory cream; topical New indication: For once-daily treatment of patients with inflammatory lesions, bumps, and pimples, of rosacea; this is the first time ever ivermectin cream is approved for use in humans; it was previously only available for use (in different form) in livestock New formulation: 1% cream for humans Trezix (new combination) (Acetaminophen, caffeine, and dihydrocodeine bitartrate; WraSer Pharmaceuticals) Classes/route: Nonopiate, nonsalicylate analgesic and antipyretic; central nervous system and cardiovascular stimulant; and semisynthetic narcotic analgesic; oral Indication: Relief of moderate-to-moderately severe pain
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Triumeq (new combination) (Abacavir, dolutegravir, and lamivudine; ViiV Healthcare) Classes/route: Integrase strand transfer inhibitor (dolu tegravir) and nucleoside analogue reverse transcriptase inhibitors (abacavir and lamivudine); oral Indication: For the treatment of patients with HIV-1 infection Velcade (new indications) (Bortezomib; Millennium Pharmaceuticals) Class/routes: Proteasome inhibitor; intravenous, subcutaneous New indications: Retreatment of adults with multiple myeloma that had previously responded to bortezomib and relapsed at least 6 months after completion of previous treatment with bortezomib; first-line treatment of newly diagnosed patients with mantle-cell lymphoma Existing indications: For the treatment of patients with multiple myeloma or with mantle-cell lymphoma Viekira Pak (new combination) See NMEs/BLAs listing Vitekta (new combination) (Elvitegravir; Gilead Sciences) Class/route: HIV-1 integrase strand transfer inhibitor; oral Indication: Used in combination with an HIV protease inhibitor coadministered with ritonavir and with other antiretroviral drug(s) for the treatment of HIV-1 infection in antiretroviral treatment–experienced adults Xgeva (new indication) (Denosumab; Amgen) Class/route: RANK ligand inhibitor; subcutaneous New indication: Treatment of hypercalcemia of malignancy refractory to bisphosphonate therapy Existing indications: Prevention of skeletal-related events
in patients with bone metastases from solid tumors, and for the treatment of adults and skeletally mature adolescents with giant-cell tumor of bone that is unresectable or where surgical resection is likely to result in severe morbidity Approval consideration: Orphan drug Xigduo XR (new combination) (Dapagliflozin and metformin hydrochloride extended release; AstraZeneca) Classes/route: SGLT2 inhibitor and biguanide; oral Indication: To improve glycemic control in adults with type 2 diabetes when treatment with dapagliflozin and with metformin is appropriate Xolair (new indication) (Omalizumab; Genentech) Class/route: Anti-immunoglobulin E antibody; subcutaneous New indication: First biologic and first medicine approved by the FDA for the treatment of chronic idiopathic urticaria in adults and adolescents aged ≥12 years who remain symptomatic despite H1 antihistamine treatment Existing indication: Treatment of moderate-to-severe persistent asthma in patients aged ≥12 years with a positive skin test or in vitro reactivity to a perennial aeroallergen and symptoms that are inadequately controlled with inhaled corticosteroids Zerbaxa (new combination) See NMEs/BLAs listing Zorvolex (new indication) (Diclofenac; Iroko Pharmaceuticals) Class/route: NSAID; oral New indication: For the management of osteoarthritis pain Existing indication: For the management of mild-to- moderate acute pain
BLA indicates biologic license application; CLL, chronic lymphocytic leukemia; DVT, deep-vein thrombosis; GLP1, glucagon-like peptide-1; HCV, hepatitis C virus; NMDA, N-methyl-D-aspartate; NME, new molecular entity; NSAID, nonsteroidal anti-inflammatory drug; PE, pulmonary embolism; REMS, Risk Evaluation and Mitigation Strategy; SGLT2, sodium-glucose cotransporter 2; VEGF, vascular endothelial growth factor.
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III. New Formulations/Dosage Forms/Dosages/Patient Population Approved in 2014 Abilify Maintena (new dosage form) (Aripiprazole; Otsuka Pharmaceutical) Class/route: Atypical antipsychotic; intramuscular New dosage form: Extended-release injectable suspension in a prefilled dual-chamber syringe Indication: For the treatment of schizophrenia
Bunavail (new formulation) (Buprenorphine and naloxone; BioDelivery Sciences) Class/route: Partial opioid agonist; oral New formulation: Buccal film Indication: For the maintenance treatment of opioid dependence
Acticlate (new dosages) (Doxycycline hyclate USP; Aqua Pharmaceuticals) Class/route: Tetracycline-class antimicrobial; oral New dosages: 75-mg and 150-mg tablets Indications: For the treatment of Rickettsial infections, sexually transmitted infections, respiratory tract infections, specific bacterial infections, ophthalmic infections, and anthrax, including inhalational anthrax (postexposure); alternative treatment for selected infections when penicillin is contraindicated; adjunctive therapy in acute intestinal amebiasis and severe acne; prophylaxis of malaria
Bydureon (new dosage form) (Exenatide; AstraZeneca) Class/route: GLP-1 receptor agonist; subcutaneous New dosage form: Extended-release formula in a 2-mg pen; already available as a single-dose tray containing a 2-mg vial Indication: As an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes mellitus
Arnuity Ellipta (new formulation) (Fluticasone furoate; GlaxoSmithKline) Class/route: Corticosteroid; oral inhalation New formulation: Once-daily oral inhalation of 100 mcg or 200 mcg using Ellipta inhaler Indication: For the once-daily maintenance treatment of asthma as prophylactic therapy in patients aged ≥12 years Auryxia (new formulation) (Ferric citrate: Keryx Biopharmaceuticals) Class/route: Phosphate binder; oral New formulation: The first and only absorbable ironbased phosphate binder proven to manage hyperphosphatemia Indication: For the control of serum phosphorus levels in patients with chronic kidney disease on dialysis Aveed (new formulation) (Testosterone undecanoate; Endo Pharmaceuticals) Class/route: Androgen; intramuscular injection New formulation: Intramuscular; already available in oral, subcutaneous, buccal, and transdermal formulations Indication: For testosterone replacement therapy in adult men for conditions associated with a deficiency or absence of endogenous testosterone, such as congenital or acquired primary hypogonadism, or congenital or acquired hypogonadotropic hypogonadism Approval consideration: REMS
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Copaxone (new dosage) (Glatiramer acetate; Teva Pharmaceutical Industries) Class/route: Immunomodulator; subcutaneous injection New dosage: Three-times weekly of 40 mg/mL, a less frequent dose than the 20 mg/mL daily that is still available for use Indication: For the treatment of patients with relapsing form of multiple sclerosis Dyloject (new dosage form) (Diclofenac; Hospira) Class/route: NSAID; intravenous New dosage form: Intravenous bolus injection over 15 seconds Indications: For use in adults for the management of mild-to-moderate pain and for the management of moderate-to-severe pain alone or in combination with opioid analgesics Embeda (new formulation) (Morphine sulfate and naltrexone hydrochloride; Pfizer) Classes/route: Opioid agonist and opioid antagonist; oral New formulation: Has properties that are expected to reduce oral abuse when the drug is crushed; capsules should be swallowed whole to avoid exposure to a potentially fatal dose of morphine Indication: For the management of pain severe enough to require daily, around-the-clock, long-term opioid treatment and for which alternative treatment options are inadequate
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Epanova (new formulation) (Omega-3-carboxylic acids; AstraZeneca) Class/route: Omega-3 fatty acid; oral New formulation: Omega-3 in free fatty acid form Indications: As an adjunct to diet to reduce triglyceride levels in adults with severe (≥500 mg/dL) hypertriglyceridemia Erwinaze (new dosage form) (Asparaginase Erwinia chrysanthemi; Jazz Pharmaceuticals) Class/routes: Antineoplastic; intramuscular, intravenous New dosage form: Intravenous Indication: As a component of a multiagent chemotherapeutic regimen for the treatment of patients with acute lymphoblastic leukemia who have developed hypersensitivity to Escherichia coli–derived asparaginase Evzio (new formulation) (Naloxone hydrochloride; Kaleo) Class/routes: Opioid antagonist; intramuscular, subcutaneous New formulation: Handheld autoinjector Indication: For the treatment of proliferating infantile hemangioma requiring systemic therapy Approval consideration: Priority review
Hemangeol (new formulation) (Propranolol hydrochloride; Pierre Fabre Dermatologie) Class/route: Beta-adrenergic blocker; oral New formulation: Oral solution for pediatric use Indication: For the treatment of proliferating infantile hemangioma requiring systemic therapy Approval consideration: Orphan drug Hysingla ER (new formulation) (Hydrocodone bitartrate; Purdue Pharma) Class/route: Opioid agonist; oral New formulation: Extended-release opioid tablet with abuse-deterrent properties that make it difficult to crush, break, or dissolve, which is intended to reduce, but not entirely prevent, abuse of the drug when taken
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Iluvien (new dosage form) (Fluocinolone acetonide intravitreal implant; Alimera Sciences) Class/route: Corticosteroid; intravitreal injection New dosage form: Single injection of the microinsert provides sustained treatment for 36 months Indication: Treatment of diabetic macular edema in patients who have been previously treated with a course of corticosteroids and did not have a clinically significant rise in intraocular pressure Incruse Ellipta (new formulation) (Umeclidinium; GlaxoSmithKline) Class/route: Anticholinergic; oral inhalation New formulation: Once-daily bronchodilator Indication: For long-term, once-daily maintenance treatment of airflow obstruction in patients with COPD
Granix (new dosage form) (Tbo-filgrastim; Teva Pharmaceutical Industries) Class/route: Leukocyte growth factor; subcutaneous New dosage form: Injection for self-administration by patients and caregivers; previously only for administration by a healthcare professional Indication: For reduction in the duration of severe neutropenia in patients with nonmyeloid malignancies receiving myelosuppressive anticancer drugs associated with a clinically significant incidence of febrile neutropenia
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orally/chewed Indication: For the management of pain severe enough to require daily, around-the-clock, long-term opioid treatment and for which alternative treatment options are inadequate Approval considerations: Priority review, REMS
Izba (new dosage) (Travoprost; Alcon Laboratories) Class/route: Prostaglandin analog; ophthalmic solution New dosage: 0.003% ophthalmic solution; already available as a 0.004% solution Indication: For reduction of elevated intraocular pressure in patients with open-angle glaucoma or ocular hypertension Kalbitor (new patient population) (Ecallantide; Dyax Corporation) Class/route: Plasma kallikrein inhibitor; subcutaneous New patient population: For the treatment of patients aged ≥12 years; previously indicated only for adults Indication: Treatment of acute attacks of hereditary angioedema in patients aged ≥12 years Kalydeco (new patient population) (Ivacaftor; Vertex Pharmaceuticals Incorporated) Class/route: CFTR; oral New patient population: Treatment of cystic fibrosis in patients aged ≥6 years who have the R117H mutation in the CFTR gene Indication: For the treatment of cystic fibrosis in patients aged ≥6 years who have 1 of the following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R [2015 new patient population: All indications were expanded to include patients aged 2-5 years]
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Kerydin (new dosage form) (Tavaborole; Anacor Pharmaceuticals) Class/route: Oxaborole antifungal; topical New dosage form: First oxaborole antifungal available as a topical solution Indication: Topical treatment of onychomycosis of the toenails from Trichophyton rubrum or Trichophyton mentagrophytes Lumizyme (new patient population) (Alglucosidase alfa; Genzyme Corporation) Class/route: Hydrolytic lysosomal glycogen-specific enzyme; intravenous New patient population: Treatment of patients with infantile-onset Pompe disease, including patients aged
