The drugs included in this review were approved by the US Food and Drug Administration (FDA) in 2015 and are grouped into the following categories:
- New Pharmaceuticals: New Molecular Entities and New Biologic License Applications
- New Combinations and New Indications
- New dosage forms and New Formulations
- New Biosimilars, Vaccines, Viral Therapies, and Blood Products
1. New Pharmaceuticals: New Molecular Entities and New Biologic License Applications
Addyi (NME)
(Flibanserin; Sprout Pharmaceuticals)
Class/route: First-in-class serotonin agonist antagonist; oral
Indication: For the treatment of premenopausal women with acquired, generalized hypoactive sexual desire disorder as characterized by low sexual desire that causes marked distress or interpersonal difficulty and is not caused by a coexisting medical or psychiatric condition, relationship problems, or the effects of a medication or other drug substance
Approval consideration: REMS program
Alecensa (NME)
(Alectinib; Genentech)
Class/route: ALK inhibitor; oral
Indication: For the treatment of patients with ALK-positive metastatic NSCLC who have progressed while receiving crizotinib or are intolerant to it
Approval consideration: Accelerated approval, breakthrough therapy, orphan drug, priority review
Aristada (NME)
(Aripiprazole lauroxil; Alkermes)
Class/route: Atypical antipsychotic, extended-release; intramuscular injection
Indication: For the treatment of adults with schizophrenia
Approval consideration: Patient Medication Guide
Avycaz (NME)
(Ceftazidime and avibactam; Cerexa)
Classes/route: Cephalosporin and beta-lactamase inhibitor; intravenous infusion
Indications: For the treatment of complicated urinary tract infections in patients aged ≥18 years; in combination with metronidazole for the treatment of complicated intra-abdominal infections in patients aged ≥18 years
Approval consideration: Fast track, priority review, QIDP
Bridion (NME)
(Sugammadex; Merck)
Class/route: First-in-class selective relaxant binding agent; intravenous
Indication: For the reversal of postsurgical neuromuscular blockade induced by rocuronium bromide and vecuronium bromide in adults
Approval consideration: Priority review
Cholbam (NME)
(Cholic acid; Asklepion Pharmaceuticals)
Class/route: Bile acid; oral
Indications: For the treatment of bile acid synthesis disorders resulting from single enzyme defects; for the adjunctive treatment of peroxisomal disorders, including Zellweger spectrum disorders, in patients with liver disease, steatorrhea, or complications from decreased fat-soluble vitamin absorption
Approval considerations: Orphan drug, priority review
Corlanor (NME)
(Ivabradine; Amgen)
Class/route: First-in-class hyperpolarization-activated cyclic nucleotide-gated channel blocker; oral
Indication: To reduce hospitalization risk from worsening heart failure in patients with stable, symptomatic chronic heart failure with left-ventricular ejection fraction ≤35% who are in sinus rhythm with a resting heart rate ≥70 bpm, and are receiving maximally tolerated doses of beta-blockers or who have a contraindication to beta-blockers
Approval considerations: Fast track, priority review
Cosentyx (BLA)
(Secukinumab; Novartis)
Class/route: First-in-class human IL-17A antagonist; subcutaneous injection
Indications: For the treatment of adults with moderate-to-severe plaque psoriasis who are candidates for systemic therapy or phototherapy; for active psoriatic arthritis; and for active ankylosing spondylitis
Cotellic (NME)
(Cobimetinib; Genentech)
Class/route: Mitogen-activated protein kinase inhibitor/extracellular signal 306 regulated kinase 1 and 2 inhibitor; oral
Indication: For the treatment of unresectable or metastatic melanoma, in combination with vemurafenib, in patients with a BRAF V600E or BRAF V600K mutation
Approval considerations: Fast track, orphan drug, priority review
Cresemba (NME)
(Isavuconazonium sulfate; Astellas Pharma)
Class/route: Azole antifungal; oral/intravenous infusion
Indications: For the treatment of invasive aspergillosis and for invasive mucormycosis
Approval considerations: Orphan drug, priority review, QIDP
Daklinza (NME)
(Daclatasvir; Bristol-Myers Squibb)
Class/route: HCV NS5A inhibitor; oral
Indication: For the treatment, in combination with sofosbuvir, of chronic HCV genotype 3 infection
Approval considerations: Fast track, priority review
Darzalex (BLA)
(Daratumumab; Janssen Biotech)
Class/route: First-in-class human CD38-directed monoclonal antibody; intravenous
Indication: For the treatment of patients with multiple myeloma who have received at least 3 previous lines of therapy including a proteasome inhibitor and an immunomodulatory agent and for those whose disease is double-refractory to a proteasome inhibitor and to an immunomodulatory agent
Approval considerations: Accelerated approval, breakthrough therapy, fast track, orphan drug, priority review
Empliciti (BLA)
(Elotuzumab; Bristol-Myers Squibb)
Class/route: First-in-class SLAMF7-directed immunostimulatory antibody; intravenous infusion
Indication: For the treatment, in combination with lenalidomide and dexamethasone, of patients with multiple myeloma who have received 1 to 3 previous therapies
Approval considerations: Breakthrough therapy, orphan drug, priority review
Entresto (NME)
(Sacubitril and valsartan; Novartis)
Class/route: First-in-class neprilysin inhibitor and angiotensin II receptor blocker; oral
Indication: For risk reduction of cardiovascular death or hospitalization caused by heart failure in patients with chronic heart failure (NYHA Class II-IV) and reduced ejection fraction
Approval considerations: Fast track, priority review
Farydak (NME)
(Panobinostat; Novartis)
Class/route: Histone deacetylase inhibitor; oral
Indication: For the treatment, in combination with bortezomib and dexamethasone, of patients with multiple myeloma who have received at least 2 previous regimens, including bortezomib and an immunomodulatory agent
Approval considerations: Accelerated approval, orphan drug, priority review, REMS program
Genvoya (NME)
(Elvitegravir, cobicistat, emtricitabine, and tenofovir alafenamide; Gilead Sciences)
Class/route: HIV-1 integrase strand transfer inhibitor, a cytochrome P3A inhibitor, and HIV-1 nucleoside analog reverse transcriptase inhibitors; oral
Indication: A complete regimen for the treatment of patients aged ≥12 years with HIV-1 infection and no antiretroviral treatment history, or to replace the current antiretroviral regimen in patients who are virologically suppressed (HIV-1 RNA <50 copies per mL) and are receiving a stable antiretroviral regimen for at least 6 months and have no history of treatment failure and no known substitutions associated with resistance to the individual components of Genvoya
Approval consideration: Fast track
Ibrance (NME)
(Palbociclib; Pfizer)
Class/route: First-in-class CDK 4 + CDK 6 inhibitor; oral
Indication: For the treatment, in combination with letrozole, of postmenopausal women with metastatic, estrogen receptor–positive, HER2-negative breast cancer as initial endocrine-based therapy
Approval considerations: Accelerated approval, breakthrough therapy, priority review
Kanuma (BLA)
(Sebelipase alfa; Alexion Pharmaceuticals)
Class/route: First-in-class hydrolytic lysosomal cholesteryl ester and triacylglycerol-specific enzyme; intravenous infusion
Indication: For the treatment of patients with lysosomal acid lipase deficiency
Approval consideration: Breakthrough therapy, fast track, orphan drug, priority review
Kengreal (NME)
(Cangrelor; The Medicines Company)
Class/route: P2Y12 platelet inhibitor; intravenous
Indication: As an adjunct to percutaneous coronary intervention for risk reduction of periprocedural myocardial infarction, repeat coronary revascularization, and stent thrombosis in patients who have not received a P2Y12 platelet inhibitor and are not receiving a glycoprotein IIb/IIIa inhibitor
Kybella (NME)
(Deoxycholic acid; Kythera Biopharmaceuticals)
Class/route: Cytolytic drug; subcutaneous injection
Indication: For improvement in the appearance of moderate-to-severe convexity or fullness associated with submental fat in adults
Lenvima (NME)
(Lenvatinib; Eisai)
Class/route: Receptor TKI; oral
Indication: Treatment of locally recurrent or metastatic, progressive, radioactive iodine-refractory differentiated thyroid cancer
Approval consideration: Orphan drug, priority review
Lonsurf (NME)
(Trifluridine and tipiracil; Taiho Oncology)
Class/route: Nucleoside metabolic inhibitor and thymidine phosphorylase inhibitor; oral
Indication: Treatment of metastatic colorectal cancer in patients who have previously received fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy, an anti-VEGF biologic therapy, and if RAS wild-type, an anti-EGFR therapy
Approval consideration: Fast track
Natpara (BLA)
(Parathyroid hormone; NPS Pharmaceuticals)
Class/route: Parathyroid hormone; subcutaneous injection
Indication: As an adjunct to calcium and vitamin D to control hypocalcemia in patients with hypoparathyroidism
Approval considerations: Orphan drug, REMS program
Ninlaro (NME)
(Ixazomib; Takeda Pharmaceuticals)
Class/route: Proteasome inhibitor; (first) oral
Indication: For the treatment, in combination with lenalidomide and dexamethasone, of patients with multiple myeloma who have received at least 1 previous therapy
Approval considerations: Orphan drug, priority review
Nucala (BLA)
(Mepolizumab; GlaxoSmithKline)
Class/route: First-in-class IL-5 antagonist monoclonal antibody; subcutaneous injection
Indication: Add-on maintenance treatment for patients aged ≥12 years with severe asthma and an eosinophilic phenotype
Odomzo (NME)
(Sonidegib; Novartis)
Class/route: Hedgehog pathway inhibitor; oral
Indication: For the treatment of adults with locally advanced basal-cell carcinoma that has recurred after surgery or radiation therapy, or those who are not candidates for surgery or radiation therapy
Orkambi (NME)
(Lumacaftor and ivacaftor; Vertex Pharmaceuticals)
Class/route: First-in-class cystic fibrosis transmembrane conductance regulator potentiator; oral
Indication: For the treatment of cystic fibrosis in patients aged ≥12 years who are homozygous for the F508del mutation in the CFTR gene (if the patient's genotype is unknown, an FDA-cleared cystic fibrosis mutation test should be used to assess the presence of the F508del mutation on both alleles of the CFTR gene)
Approval considerations: Breakthrough therapy, fast track, orphan drug, priority review
Portrazza (BLA)
(Necitumumab; Eli Lilly)
Class/route: EGFR antagonist; intravenous infusion
Indication: For the first-line treatment, in combination with gemcitabine and cisplatin, of patients with metastatic squamous NSCLC
Approval considerations: Fast track, orphan drug
Praluent (BLA)
(Alirocumab; sanofi-aventis)
Class/route: First-in-class PCSK9 inhibitor antibody; subcutaneous injection
Indication: As an adjunct to diet and maximally tolerated statin therapy for adults with heterozygous familial hypercholesterolemia or with clinical atherosclerotic CVD who require additional LDL-C lowering
Praxbind (BLA)
(Idarucizumab; Boehringer Ingelheim)
Class/route: First-in-class humanized monoclonal antibody fragment; intravenous infusion
Indication: For reversal of dabigatran anticoagulant effects in patients requiring emergency surgery or urgent procedures or in those with life-threatening or uncontrolled bleeding
Approval considerations: Accelerated approval, breakthrough therapy, orphan drug, priority review
Repatha (BLA)
(Evolocumab; Amgen)
Class/route: PCSK9 inhibitor antibody; intravenous infusion
Indication: For the treatment of heterozygous familial hypercholesterolemia or clinical atherosclerotic CVD, as an adjunct to diet and maximally tolerated statin therapy in adults who require additional LDL-C lowering; and as an adjunct to diet and other LDL-lowering therapies (eg, statins, ezetimibe, LDL apheresis) in patients with homozygous familial hypercholesterolemia who require additional lowering of LDL-C
Approval consideration: Orphan drug for the treatment of homozygous familial hypercholesterolemia
Rexulti (NME)
(Brexpiprazole; Otsuka Pharmaceutical Company)
Class/route: Atypical antipsychotic; oral
Indication: For the treatment of major depressive disorder as an adjunctive therapy to antidepressants; and for the treatment of schizophrenia
Savaysa (NME)
(Edoxaban; Daiichi Sankyo)
Class/route: Factor Xa inhibitor; oral
Indication: To reduce the risk for stroke and systemic embolism in patients with nonvalvular atrial fibrillation; and for the treatment of deep-vein thrombosis and pulmonary embolism after 5 to 10 days of initial therapy with a parenteral anticoagulant
Strensiq (BLA)
(Asfotase alfa; Alexion Pharmaceuticals)
Class/route: First-in-class tissue-nonspecific alkaline phosphatase; subcutaneous injection
Indication: For the treatment of perinatal or infantile-onset and juvenile-onset hypophosphatasia
Approval considerations: Breakthrough therapy, fast track, orphan drug, priority review
Tagrisso (NME)
(Osimertinib; AstraZeneca)
Class/route: EGFR kinase inhibitor; oral
Indication: For the treatment of metastatic EGFR T790M mutation—positive NSCLC (as detected by an FDA-approved test) whose disease progressed while or after receiving EGFR TKI therapy
Approval considerations: Accelerated approval, breakthrough therapy, fast track, orphan drug, priority review
Tresiba (NME)
(Insulin degludec injection; Novo Nordisk)
Class/route: Long-acting human insulin analog; subcutaneous injection
Indication: To improve glycemic control in adults with diabetes mellitus
Unituxin (BLA)
(Dinutuximab; United Therapeutics)
Class/route: First-in-class GD2-binding monoclonal antibody; intravenous infusion
Indication: For the treatment, in combination with granulocyte-macrophage colony-stimulating factor, IL-2, and 13-cis-retinoic acid, of pediatric patients with high-risk neuroblastoma who achieve at least a partial response to previous first-line multiagent, multimodality therapy
Approval considerations: Orphan drug, priority review
Uptravi (NME)
(Selexipag; Actelion Pharmaceuticals)
Class/route: Prostacyclin receptor agonist; oral
Indication: For the treatment of pulmonary arterial hypertension (WHO Group I) to delay disease progression and reduce hospitalization risk
Approval consideration: Orphan drug
Varubi (NME)
(Rolapitant; Tesaro)
Class/route: Substance P/neurokinin 1 receptor antagonist; oral
Indication: For the prevention, in combination with other antiemetic agents, of delayed nausea and vomiting associated with initial and repeat courses of emetogenic chemotherapy for cancer, including, but not limited to, highly emetogenic chemotherapy
Veltassa (NME)
(Patiromer; Relypsa)
Class/route: Potassium binder; oral
Indication: For the treatment of hyperkalemia in adults
Viberzi (NME)
(Eluxadoline; Forest Pharmaceuticals)
Class/route: Mu-opioid receptor agonist; oral
Indication: For the treatment of irritable bowel syndrome and diarrhea in adults
Approval considerations: Fast track, priority review
Vraylar (NME)
(Cariprazine; Actavis Pharma)
Class/route: Atypical antipsychotic; oral
Indication: For the treatment of schizophrenia; and for acute treatment of manic or mixed episodes associated with bipolar I disorder
Xuriden (NME)
(Uridine triacetate; Wellstat Therapeutics Corporation)
Class/route: First-in-class pyrimidine analog for uridine replacement; oral
Indication: For the treatment of hereditary orotic aciduria
Approval considerations: Breakthrough therapy, orphan drug, priority review
Yondelis (NME)
(Trabectedin; Janssen Products)
Class/route: Alkylating drug; intravenous infusion
Indication: For the treatment of unresectable or metastatic liposarcoma or leiomyosarcoma in patients who received a previous anthracycline-containing regimen
Approval considerations: Orphan drug, priority review
Zurampic (NME)
(Lesinurad; Ardea Biosciences)
Class/route: URAT1 inhibitor; oral
Indication: For treatment, in combination with a xanthine oxidase inhibitor, of hyperuricemia associated with gout in patients who have not achieved target serum uric acid levels with a xanthine oxidase inhibitor alone
ALK indicates anaplastic lymphoma kinase; bpm, beats per minute; BLA, biologic license application; CDK, cyclin-dependent kinase; CVD, cardiovascular disease; EGFR, epidermal growth factor receptor; FDA, US Food and Drug Administration; HCV, hepatitis C virus; IL, interleukin; LDL-C, low-density lipoprotein cholesterol; NME, new molecular entity; NSCLC, non—small-cell lung cancer; NYHA, New York Heart Association; PCSK9, proprotein convertase subtilisin kexin type 9; QIDP, Qualified Infectious Disease Product; REMS, Risk Evaluation and Mitigation Strategy; TKI, tyrosine kinase inhibitor; URAT1, urate transporter 1; VEGF, vascular endothelial growth factor; WHO, World Health Organization.
2. New Combinations and New Indications
Aptiom (new indication)
(Eslicarbazepine acetate; Sunovion Pharmaceuticals)
Class/route: Anticonvulsant; oral
New indication: For the treatment of partial-onset seizures as monotherapy
Existing indication: For the treatment of partial-onset seizures as adjunctive therapy
Avelox (new indication)
(Moxifloxacin; Bayer HealthCare)
Class/route: Fluoroquinolone antibacterial; oral and intravenous
New indication: For the treatment of plague in adults aged ≥18 years
Existing indication: In adults aged ≥18 years, for the treatment of acute bacterial sinusitis; for the treatment of acute bacterial exacerbation of chronic bronchitis; for the treatment of community-acquired pneumonia; for the treatment of uncomplicated and complicated skin and skin structure infections; and for the treatment of complicated intra-abdominal infections
Avycaz (new combination) See NMEs/BLAs listing
Banzel (new indication)
(Rufinamide; Eisai)
Class/route: Carboxamide; oral
New indication: For the adjunctive treatment of seizures associated with Lennox-Gastaut syndrome in patients aged ≥1 years
Existing indication: For the adjunctive treatment of seizures associated with Lennox-Gastaut syndrome in patients aged ≥4 years
BioThrax (new indication)
(Anthrax vaccine adsorbed; Emergent BioDefense Operations Lansing)
Class/route: Vaccine; intramuscular, subcutaneous
New indication: For the pre- and postexposure prophylaxis of disease after suspected or confirmed Bacillus anthracis exposure, when administered in conjunction with recommended antibacterial drugs
Existing indication: For the pre-exposure prophylaxis of anthrax in people who are at high risk for exposure
Breo Ellipta (new indication)
(Fluticasone furoate and vilanterol; GlaxoSmithKline and Theravance)
Class/route: Inhaled corticosteroid and long-acting beta2-adrenergic agonist; oral inhalation
New indication: For the once-daily treatment of asthma in patients aged ≥18 years
Existing indication: For the long-term, once-daily maintenance treatment of airflow obstruction and for reducing exacerbations in patients with COPD
Brilinta (new indication)
(Ticagrelor; AstraZeneca)
Class/route: P2Y12 platelet inhibitor; oral
New indication: To reduce the rate of CV death, MI, and stroke in patients with ACS or a history of MI; to reduce the rate of stent thrombosis in patients who have been stented for the treatment of ACS
Existing indication: To reduce the rate of thrombotic CV events in patients with ACS
Approval consideration: Priority review.
See also New Dosage, New dosage form listing
Cyramza (new indication)
(Ramucirumab; Eli Lilly)
Class/route: Human VEGF receptor-2 antagonist; intravenous
New indication: For the treatment, in combination with FOLFIRI, of metastatic colorectal cancer in patients whose disease progressed during or after previous therapy with bevacizumab, oxaliplatin, and a fluoropyrimidine
Existing indication: 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 NSCLC in patients whose disease progressed during or after platinum based chemotherapy
Dysport (new indication)
(AbobotulinumtoxinA; Ipsen)
Classes/route: Acetylcholine release inhibitor and neuromuscular-blocking agent; intramuscular
New indication: Treatment of upper limb spasticity in adults
Existing indication: For the treatment of adults with cervical dystonia; for the temporary improvement in the appearance of moderate-to-severe glabellar lines associated with procerus and corrugator muscle activity in adults aged <65 years
Emend (new indications)
(Aprepitant; Merck)
Class/route: Substance P/neurokinin 1 receptor antagonist; oral
New indication: In combination with other antiemetic agents, in patients aged ≥12 years and in patients aged <12 years who weigh ≥30 kg for the prevention of acute and delayed nausea and vomiting associated with initial and repeat courses of highly emetogenic cancer chemotherapy; for the treatment of nausea and vomiting associated with initial and repeat courses of moderately emetogenic chemotherapy
Existing indication: In adults, in combination with other antiemetic agents, for the prevention of acute and delayed nausea and vomiting associated with initial and repeat courses of highly emetogenic chemotherapy; for the treatment of nausea and vomiting associated with initial and repeat courses of moderately emetogenic chemotherapy; for the prevention of postoperative nausea and vomiting in adults
Entresto (new combination) See NMEs/BLAs listing
Eylea (new indication)
(Aflibercept; Regeneron Pharmaceuticals)
Class/route: VEGF inhibitor; intravitreal injection
New indication: Diabetic retinopathy in patients with diabetic macular edema
Existing indication: For the treatment of neovascular (wet) age-related macular degeneration; for the treatment of macular edema after retinal vein occlusion; treatment of diabetic macular edema
Approval considerations: Breakthrough therapy, priority review
Fycompa (new indication)
(Perampanel; Eisai)
Class/route: Noncompetitive AMPA glutamate receptor antagonist; oral
New indication: As adjunctive therapy for the treatment of primary generalized tonic-clonic seizures in patients with epilepsy aged ≥12 years
Existing indication: As adjunctive therapy for the treatment of partial-onset seizures with or without secondarily generalized seizures in patients with epilepsy aged ≥12 years
Gardasil 9 (new indication)
(HPV 9-valent vaccine, recombinant; Merck)
Class/route: Vaccine; intramuscular
New indication: In males aged 16 through 26 years, for the prevention of anal cancer caused by HPV types 16, 18, 31, 33, 45, 52, and 58; for the treatment of precancerous or dysplastic lesions caused by HPV types 6, 11, 16, 18, 31, 33, 45, 52, and 58; and for the treatment of genital warts caused by HPV types 6 and 11
Existing indication: In girls and women aged 9 through 26 years, for the prevention of cervical, vulvar, vaginal, and anal cancer caused by HPV types 16, 18, 31, 33, 45, 52, and 58; genital warts (condyloma acuminata) caused by HPV types 6 and 11; types 6, 11, 16, 18, 31, 33, 45, 52, and 58 precancerous or dysplastic lesions caused by HPV; cervical intraepithelial neoplasia grade 2/3 and cervical adenocarcinoma in situ; cervical intraepithelial neoplasia grade 1; vulvar intraepithelial neoplasia grades 2 and 3; vaginal intraepithelial neoplasia grades 2 and 3; and anal intraepithelial neoplasia grades 1, 2, and 3; in boys and men aged 9 through 15 years, for the prevention of anal cancer caused by HPV types 16, 18, 31, 33, 45, 52, and 58; genital warts (condyloma acuminata) caused by HPV types 6 and 11; types 6, 11, 16, 18, 31, 33, 45, 52, and 58 precancerous or dysplastic lesions caused by HPV; anal intraepithelial neoplasia grades 1, 2, and 3
Genvoya (new combination) See NMEs/BLAs listing
Harvoni (new indication)
(Ledipasvir and sofosbuvir; Gilead Sciences)
Class/route: An HCV NS5A inhibitor and HCV nucleotide analog NS5B polymerase inhibitor; oral
New indication: For the treatment, with or without ribavirin, of chronic HCV genotypes 4, 5, or 6 infection and in patients coinfected with HIV
Existing indication: For the treatment, with or without ribavirin, of chronic HCV genotype 1
Humira (new indication)
(Adalimumab; AbbVie)
Class/route: Tumor necrosis factor blocker; subcutaneous
New indication: For the treatment of moderate-to-severe hidradenitis suppurativa
Existing indication: For the treatment of rheumatoid arthritis; juvenile idiopathic arthritis; psoriatic arthritis; ankylosing spondylitis; Crohn's disease in adults and in pediatric patients aged ≥6 years; ulcerative colitis; and plaque psoriasis
Approval consideration: Orphan drug
Imbruvica (new indication)
(Ibrutinib; Janssen Biotech)
Class/route: Bruton's TKI; oral
New indication: First drug approved for the treatment of patients with Waldenström's macroglobulinemia
Existing indication: For the treatment of CLL in patients who have received at least 1 previous therapy; for the treatment of mantle-cell lymphoma in patients who have received at least 1 previous therapy; for the treatment of patients with CLL with 17p deletion
Iressa (new indication)
(Gefitinib; AstraZeneca)
Class/route: TKI; oral
New indication: For the first-line treatment of metastatic NSCLC in patients whose tumors have EGFR exon 19 deletions or exon 21 (L858R) substitution mutations as detected by an FDA-approved test
Existing indication: For the third-line treatment of advanced NSCLC after nonresponse to chemotherapy
Kalydeco (new indication)
(Ivacaftor; Vertex Pharmaceuticals)
Class/route: Cystic fibrosis transmembrane conductance regulator potentiator; oral
New indication: For the treatment of cystic fibrosis in patients aged ≥2 years who have G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, or R117H mutations in the CFTR gene
Existing indication: For the treatment of cystic fibrosis in patients aged ≥6 years who have G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, or R117H mutations in the CFTR gene
Keytruda (new indications)
(Pembrolizumab; Merck)
Class/route: PD-1—blocking antibody; intravenous infusion
New Indications: For the treatment of metastatic NSCLC in patients whose tumors express PD-L1 as determined by a genetic diagnostic test, and whose disease progressed with or after platinum-based chemotherapy; for the first-line treatment of unresectable or metastatic melanoma
Existing indication: For the treatment of unresectable or metastatic melanoma
Approval consideration: Accelerated approval
Kyprolis (new indication)
(Carfilzomib; Amgen)
Class/route: Proteasome inhibitor; intravenous infusion
New indication: In combination with lenalidomide and dexamethasone, for the treatment of multiple myeloma in patients who received 1 to 3 previous lines of therapy
Existing indication: As monotherapy for relapsed or refractory multiple myeloma in patients who received at least 2 previous therapies, including bortezomib and an immunomodulatory agent, and whose disease progressed during or within 60 days after completing the previous therapy
Letairis plus tadalafil (new combination)
(Ambrisentran and tadalafil; Gilead Sciences)
Class/route: Endothelin receptor antagonist and phosphodiesterase type 5 inhibitor; oral
Indications: For the treatment of PAH (WHO Group 1) to reduce the risks for disease progression and hospitalization for worsening PAH and to improve exercise ability
Approval Considerations: Orphan drug, REMS program
Lonsurf (new combination) See NMEs/BLAs listing
Lucentis (new indication)
(Ranibizumab; Genentech)
Class/route: VEGF inhibitor; intravitreal injection
New indication: Treatment of diabetic retinopathy in patients with diabetic macular edema
Existing indication: Treatment of neovascular (wet) age-related macular degeneration; treatment of macular edema after retinal vein occlusion; treatment of diabetic macular edema
Opdivo (new indications)
(Nivolumab; Bristol-Myers Squibb)
Class/route: PD-1—blocking antibody; intravenous infusion
New indication: For the treatment of metastatic squamous NSCLC that progressed with or after platinum-based chemotherapy; metastatic NSCLC that progressed with or after platinum-based chemotherapy; and advanced renal-cell carcinoma in patients who received previous antiangiogenic therapy
Existing indication: For the treatment of BRAF V600 mutation1—positive unresectable or metastatic melanoma, as a single agent; and for the treatment of unresectable or metastatic melanoma and disease progression after ipilimumab therapy, and, if BRAF V600 mutation positive, a BRAF inhibitor
Approval considerations: Accelerated approval, breakthrough therapy, fast track, priority review
Opdivo plus Yervoy (new combination)
(Nivolumab and ipilimumab; Bristol-Myers Squibb)
Class/route: PD-1—blocking antibody and human CTLA-4—blocking antibody; intravenous
Indication: In combination with ipilimumab as a treatment for patients with BRAF V600 wild-type and BRAF V600 mutation—positive unresectable or metastatic melanoma; in January 2016, the FDA approved an expanded indication to include patients with advanced melanoma, regardless of their BRAF mutational status
Approval consideration: Accelerated approval
Orkambi (new combination) See NMEs/BLAs listing
OxyContin (new indication)
(Oxycodone; Purdue Pharma)
Class/route: Opioid analgesic; oral
New indication: For the treatment of pain severe enough to require daily, around-the-clock, long-term opioid treatment and for which alternative treatment options are inadequate in opioid-tolerant pediatric patients aged ≥11 years who are already receiving and tolerate a minimum daily opioid dose of ≥20 mg oxycodone orally or its equivalent
Existing indication: For the treatment of pain severe enough to require daily, around-the-clock, long-term opioid treatment and for which alternative treatment options are inadequate in opioid-tolerant pediatric patients aged ≥16 years who are already receiving and tolerate a minimum daily opioid dose of at least 20 mg oxycodone orally or its equivalent
Pomalyst (new indication)
(Pomalidomide; Celgene)
Class/route: Thalidomide analog; oral
New indication: In combination with dexamethasone, for the treatment of patients with multiple myeloma who have received at least 2 previous therapies including lenalidomide and a proteasome inhibitor and have demonstrated disease progression on or within 60 days of completion of their last therapy
Existing indication: For the treatment of patients with multiple myeloma who have received at least 2 previous therapies, including lenalidomide and bortezomib, and whose disease progressed during or within 60 days of completion of their last therapy
Approval considerations: Orphan drug, REMS program
Pradaxa (new indication)
(Dabigatran etexilate mesylate; Boehringer Ingelheim)
Class/route: Direct thrombin inhibitor; oral
New indication: For the prophylaxis of DVT and PE in patients who have undergone hip replacement surgery
Existing indication: To reduce the risks for stroke and systemic embolism in patients with nonvalvular atrial fibrillation; for the treatment of DVT and PE in patients who have been treated with a parenteral anticoagulant for 5 to 10 days; to reduce the risk for recurrence of DVT and PE in patients who have been previously treated
Prestalia (new combination)
(Perindopril arginine and amlodipine; Symplmed Pharmaceuticals)
Class/route: Angiotensin-converting enzyme inhibitor and dihydropyridine calcium channel blocker; oral
Indications: For the treatment of hypertension to lower blood pressure in patients not adequately controlled with monotherapy; and as initial therapy in patients likely to need multiple drugs
Prezcobix (new combination)
(Darunavir and cobicistat; Janssen)
Class/route: HIV-1 protease inhibitor and cytochrome P3A inhibitor; oral
Indication: For the treatment of HIV-1 infection in adults
Procysbi (new indication)
(Cysteamine bitartrate; Raptor Pharmaceutical Co.)
Class/route: Cystine-depleting agent; oral
New indication: For the treatment of nephropathic cystinosis in pediatric patients aged ≥2 years
Existing indication: For the treatment of nephropathic cystinosis in adults and in pediatric patients ≥6 years
Promacta (new indications)
(Eltrombopag; Ligand Pharmaceuticals)
Class/route: Thrombopoietin receptor agonist; oral
New indication: For the treatment of thrombocytopenia in pediatric patients aged ≥6 years with chronic immune (idiopathic) thrombocytopenia who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy, which was subsequently expanded to include pediatric patients aged ≥1 years
Existing indication: For the treatment of thrombocytopenia in adults with chronic immune (idiopathic) thrombocytopenia who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy; for the treatment of thrombocytopenia in patients with chronic hepatitis C to allow the initiation and maintenance of interferon-based therapy; for the treatment of patients with severe aplastic anemia who have had an insufficient response to immunosuppressive therapy
Approval consideration: Orphan drug
Qudexy XR (new indication)
(Topiramate; Upsher-Smith Laboratories)
Class/route: Carbonic anhydrase inhibitor anticonvulsant; oral
New indication: For initial monotherapy and as adjunctive therapy in patients aged ≥2 years with partial-onset or primary generalized tonic-clonic seizures
Existing indication: For initial monotherapy and as adjunctive therapy in patients aged ≥10 years with partial-onset or primary generalized tonic-clonic seizures; adjunctive therapy in patients aged ≥2 years with seizures associated with Lennox-Gastaut syndrome
Rapamune (new indication)
(Sirolimus; Pfizer)
Class/route: Immunosuppressant; oral
New indication: For the treatment of lymphangioleiomyomatosis
Existing indication: For the prophylaxis of organ rejection in patients aged ≥13 years who are receiving renal transplants
Revlimid (new indication)
(Lenalidomide; Celgene)
Class/route: Thalidomide analog; oral
New indication: For the first-line treatment, in combination with dexamethasone, of all patients with multiple myeloma, including newly diagnosed patients
Existing indication: For the treatment of multiple myeloma, in combination with dexamethasone, in patients who have received at least 1 previous therapy; for the treatment of patients with transfusion-dependent anemia resulting from low- or intermediate-1—risk myelodysplastic syndromes associated with a deletion 5q cytogenetic abnormality with or without additional cytogenetic abnormalities; for the treatment of patients with mantle-cell lymphoma that has relapsed or progressed after 2 previous therapies, including bortezomib
Approval consideration: REMS program
Saphris (new indication)
(Asenapine; Actavis)
Class/route: Atypical antipsychotic; oral
New indication: As monotherapy for the acute treatment of manic or mixed episodes associated with bipolar I disorder in pediatric patients aged 10 to 17 years
Existing indication: For the treatment of schizophrenia; for the acute treatment of manic or mixed episodes associated with bipolar I disorder as monotherapy or adjunctive treatment to lithium or valproate
Spiriva Respimat (new indication)
(Tiotropium bromide; Boehringer Ingelheim)
Class/route: Anticholinergic; oral inhalation
New indication: For the long-term, once-daily maintenance treatment of asthma in patients aged ≥12 years
Existing indication: For the long-term, once-daily maintenance treatment of bronchospasm associated with COPD, and for reducing COPD exacerbations
Stiolto Respimat (new combination)
(Tiotropium bromide and olodaterol; Boehringer Ingelheim)
Class/route: Anticholinergic and long-acting beta2-adrenergic agonist; oral inhalation
Indication: For the long-term, once-daily maintenance treatment of airflow obstruction in patients with COPD
Synjardy (new combination)
(Empagliflozin and metformin hydrochloride; Boehringer Ingelheim)
Class/route: Sodium-glucose co-transporter 2 inhibitor and biguanide; oral
Indication: As an adjunct to diet and exercise, to improve glycemic control in adults with type 2 diabetes that is not adequately controlled with a regimen containing empagliflozin or metformin, or in patients already receiving empagliflozin and metformin therapy
Technivie (new combination)
(Ombitasvir, paritaprevir, and ritonavir; AbbVie)
Class/route: HCV NS5A inhibitor, HCV NS3/4A protease inhibitor, and cytochrome P3A inhibitor; oral
New Combination: In combination with ribavirin, this is the first drug to demonstrate safety and efficacy in the treatment of genotype 4 HCV infections without the need for the coadministration of interferon
Indication: For the treatment, in combination with ribavirin, of patients with genotype 4 chronic HCV infection without cirrhosis
Approval consideration: Priority review
Treximet (new combination)
(Sumatriptan and naproxen sodium; Pernix Therapeutics)
Class/route: Serotonin 5-HT1B/1D receptor agonist and a nonsteroidal anti-inflammatory drug; oral
Indication: For the acute treatment of migraine with or without aura in patients aged ≥12 years
Utibron Neohaler (new combination)
(Indacaterol and glycopyrrolate; Novartis)
Class/route: Long-acting beta2-adrenergic agonist and anticholinergic; oral inhalation
Indication: For the long-term maintenance treatment of airflow obstruction in patients with COPD
Vistogard (new indication)
(Uridine triacetate; Wellstat Therapeutics)
Class/route: Pyrimidine analog; oral
New indications: For the emergency treatment of adults and pediatric patients after a fluorouracil or a capecitabine overdose regardless of symptoms; for the emergency treatment of adults and pediatric patients who exhibit early-onset, severe, or life-threatening toxicity affecting the cardiac or central nervous system and/or early-onset, unusually severe adverse reactions within 96 hours after receiving fluorouracil or capecitabine therapy
Existing indication: For use as an antidote in the treatment of 5-fluorouracil poisoning
Approval considerations: Fast track, orphan drug, priority review
Vyvanse (new indication)
(Lisdexamfetamine dimesylate; Shire)
Class/route: Central nervous system stimulant; oral
New indication: For the treatment of moderate-to-severe binge-eating disorder
Existing indication: For the treatment of patients aged >6 years with attention-deficit/hyperactivity disorder
Approval consideration: Priority review
Xeomin (new indication)
(IncobotulinumtoxinA; Merz North America)
Classes/route: Acetylcholine release inhibitor and neuromuscular-blocking agent; intramuscular
New indication: For the treatment or improvement of adults with upper limb spasticity
Existing indications: For the treatment or improvement of adults with cervical dystonia; for the treatment or improvement of adults with blepharospasm with previous onabotulinumtoxinA treatment; for the temporary improvement in the appearance of moderate-to-severe glabellar lines with corrugator and/or procerus muscle activity
Xifaxan (new indication)
(Rifaximin; Salix Pharmaceuticals)
Class/route: Rifamycin antibacterial; oral
New indications: For the treatment of irritable bowel syndrome with diarrhea in adults
Existing indications: For the treatment of travelers' diarrhea caused by noninvasive strains of Escherichia coli in patients aged ≥12 years; for reducing the risk for overt hepatic encephalopathy recurrence in adults
Yervoy (new indications; new combination)
(Ipilimumab; Bristol-Myers Squibb)
Class/route: Human CTLA-4—blocking antibody; intravenous
New indications: For the adjuvant treatment of patients with cutaneous melanoma and pathologic involvement of regional lymph nodes of >1 mm who have undergone complete resection, including total lymphadenectomy; and as adjuvant therapy for patients with stage III melanoma, to lower the risk for melanoma recurrence after surgery
New combinations: See listing for Opdivo plus Yervoy
Existing indication: For the treatment of unresectable or metastatic melanoma
Approval consideration: Accelerated approval
Zomig (new indication)
(Zolmitriptan; Impax Specialty Pharma)
Class/route: Serotonin 5-HT1B/1D receptor agonist; nasal spray
New indication: For the acute treatment of migraine with or without aura in pediatric patients aged ≥12 years
Existing indication: For the acute treatment of migraine with or without aura in adults
Zubsolv (new indication)
(Buprenorphine and naloxone; Orexo)
Class/route: Partial opioid agonist; sublingual
New indication: For the induction of buprenorphine maintenance therapy in patients with opioid dependence
Existing indication: For the treatment of opioid dependence
ACS indicates acute coronary syndrome; BLA, biologic license application; CLL, chronic lymphocytic leukemia; COPD, chronic obstructive pulmonary disease; CTLA-4, cytotoxic T-lymphocyte antigen 4; CV, cardiovascular; DVT, deep-vein thrombosis; EGFR, epidermal growth factor receptor; FDA, US Food and Drug Administration; FOLFIRI, folinic acid, fluorouracil, irinotecan; HCV, hepatitis C virus; HPV, human papillomavirus; MI, myocardial infarction; NME, new molecular entity; NSCLC, non—small-cell lung cancer; PAH, pulmonary arterial hypertension; PD-1, programmed death receptor-1; PD-L1, PD ligand 1; PE, pulmonary embolism; REMS, Risk Evaluation and Mitigation Strategy; TKI, tyrosine kinase inhibitor; VEGF, vascular endothelial growth factor; WHO, World Health Organization.
3. New Dosages, New dosage forms, New Formulations, and New Delivery Systems
Aggrastat (new dosage)
(Tirofiban hydrochloride; Medicure)
Class/route: Platelet aggregation inhibitor; intravenous
New dosage: Infusion duration for delivery of the bolus within 5 minutes
Existing dosage: Infusion duration for delivery of the bolus over 3 minutes
Indication: For reduction of the rate of thrombotic cardiovascular events (combined end point of death, MI, or refractory ischemia/repeat cardiac procedure) in patients with non-ST elevation ACS
Belbuca (new dosage form)
(Buprenorphine; Endo Pharmaceuticals)
Class/route: Partial opioid agonist; sublingual
New dosage form: Buccal film
Existing dosage forms: Intramuscular; intravenous; transdermal; intranasal; rectal; oral
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 consideration: REMS program
Bendeka (new formulation)
(Bendamustine hydrochloride; Teva/Eagle Pharmaceuticals)
Class/route: ALkylating drug; intravenous infusion
New formulation : A liquid, low-volume (50-mL), short-infusion-time (10-minute) formulation
Existing formulation: 500-mL admixture in a 60-minute intravenous infusion
Indication: For the treatment of chronic lymphocytic leukemia and indolent B-cell non-Hodgkin lymphoma that has progressed during or within 6 months of treatment with rituximab or a rituximab-containing regimen
Betaconnect (new delivery system)
(Interferon beta-1b; Bayer HealthCare)
Class/route: Interferon beta; subcutaneous
New delivery system: First electronic autoinjector
Existing delivery system: Self-administered subcutaneous injection using a prefilled syringe
Indication: For the treatment of relapsing forms of multiple sclerosis to reduce the frequency of clinical exacerbations
Brilinta (new dosage/new dosage form)
(Ticagrelor; AstraZeneca)
Class/route: P2Y12 platelet inhibitor; oral
New dosage/dosage form: 60-mg tablets; in patients who are unable to swallow tablets whole, the drug may be crushed and administered in water by swallowing or by nasogastric tube
Existing dosage: 90-mg tablets
Indications: To reduce the rate of cardiovascular death and stroke in patients with ACS or a history of MI; to reduce the rate of stent thrombosis in patients who have been stented for the treatment of ACS
Approval consideration: Priority review
See also New Indication
Duopa (new dosage form)
Carbidopa and levodopa; AbbVie)
Class/route: Aromatic amino acid decarboxylation inhibitor and aromatic amino acid; enteral suspension
New dosage form: Enteral suspension placed directly into the small intestine via a tube inserted by a percutaneous endoscopic gastrostomy procedure with jejunal extension that provides 16 continuous hours of the drug
Existing dosage form: Immediate-release oral tablet
Indication: For the treatment of motor fluctuations in patients with advanced Parkinson's disease
Approval consideration: Orphan drug
Enstilar (new dosage form)
(Calcipotriene and betamethasone dipropionate; LEO Pharma)
Class/route: Vitamin D analog and corticosteroid; topical
New dosage form: 0.005%/0.064% topical foam
Existing dosage form: Topical gel
Indication: For the topical treatment of plaque psoriasis in patients aged ≥18 years
Envarsus XR (new dosage form)
(Tacrolimus extended-release tablets; Veloxis)
Class/route: Calcineurin-inhibitor immunosuppressant; oral
New dosage form: Once-daily extended-release tablet
Existing dosage forms: Twice-daily immediate-release capsule; intravenous injection
Indication: For the prophylaxis of organ rejection in patients having a kidney transplant who converted from tacrolimus immediate-release formulations in combination with other immunosuppressants
Finacia foam (new dosage form)
(Azelaic acid; Bayer HealthCare Pharmaceuticals)
Class/route: Saturated dicarboxylic acid; topical
New dosage form: Topical foam
Existing dosage form: Topical cream
Indication: For the topical treatment of inflammatory papules and pustules of mild-to-moderate rosacea
Kalydeco (new dosage form)
(Ivacaftor; Vertex Pharmaceuticals)
Class/route: Cystic fibrosis transmembrane conductance regulator potentiator; oral
New dosage form: Oral granules
Existing dosage form: Oral tablets
Indication: For the treatment of cystic fibrosis in patients aged ≥2 years who have G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, or R117H mutations in the CFTR gene
Minocin for Injection (new formulation)
(Minocycline; The Medicines Company)
Class/route: Tetracycline antibiotic; intravenous injection
New formulation: Contains magnesium sulfate as an inactive excipient in a lower fluid volume, which allows for a wider range of infusion volumes (100-1000 mL)
Indications: For the treatment of infections resulting from susceptible isolates of designated microorganisms, including Acinetobacter species bacteria; for the treatment of infections caused by certain gram-negative or gram-positive bacteria when bacteriologic testing indicates appropriate susceptibility to the drug
Approval considerations: Priority review, QIDP
Onivyde (new formulation)
(Irinotecan liposome injection; Merrimack Pharmaceuticals)
Class/route: Topoisomerase inhibitor; intravenous injection
New formulation: Encapsulated irinotecan in a liposomal formulation
Existing formulation: Irinotecan hydrochloride injection
Indication: In combination with leucovorin and 5-fluorouracil, for the treatment of advanced or metastatic pancreatic cancer that progressed after gemcitabine-based chemotherapy
Approval considerations: Priority review, orphan drug
Onsolis (new formulation)
(Fentanyl buccal soluble film; BioDelivery Sciences)
Class/route: Opioid agonist; buccal
New formulation: Fade-proof pink color
Indications: For the management of breakthrough pain in patients with cancer aged ≥18 years who are already receiving and who are tolerant to opioid therapy for their underlying persistent cancer pain
Approval consideration: REMS program
Otiprio (new formulation)
(Ciprofloxacin otic suspension; Otonomy)
Class/route: Fluoroquinolone antibacterial; intratympanic suspension
New formulation: A thermosensitive gel with drug microparticles to enable single-dose treatment
Existing formulation: Immediate-release tablets; extended-release tablets; oral suspension; intravenous infusion; ophthalmic ointment and solution; otic solution
Indication: For the treatment of pediatric patients with bilateral otitis media with effusion undergoing tympanostomy tube placement
ProAir RespiClick (new dosage form)
(Albuterol sulfate; Teva)
Class/route: Beta2-adrenergic agonist; oral
New dosage form: Breath-actuated, dry-powder rescue inhaler
Existing dosage form: Inhalation aerosol
Indications: For the treatment or prevention of bronchospasm in patients with a reversible obstructive airway disease; and for the prevention of exercise-induced bronchospasm in patients aged ≥12 years
Rytary (new formulation)
(Carbidopa and levodopa; Impax Laboratories)
Class/route: Aromatic amino acid decarboxylation inhibitor and aromatic amino acid; oral
New Formulation: Extended-release oral capsule
Existing dosage form: Immediate-release oral tablet
Indications: For the treatment of Parkinson's disease, postencephalitic parkinsonism, and parkinsonism that may follow carbon monoxide intoxication or manganese intoxication
Toujeo U-300 (new formulation)
(Insulin glargine injection; sanofi-aventis)
Class/route: Long-acting human insulin analog; subcutaneous injection
New Formulation: Administered as 300 units/mL of insulin glargine in a 1.5-mL SoloStar disposable prefilled pen
Existing Formulation: 100 units/mL of insulin glargine
Indication: To improve glycemic control in adults with diabetes mellitus
Viibryd (new dosage)
(Vilazodone hydrochloride; Actavis)
Classes/route: Selective serotonin reuptake inhibitor and 5-HT1A receptor partial agonist; oral
New dosage: 20-mg dose
Existing dosage: 40-mg dose
Indication: For the treatment of major depressive disorder
Vivlodex (new dosage form)
(Meloxicam; Iroko Pharmaceuticals)
Class/route: Nonsteroidal anti-inflammatory drug; oral
New dosage form: Capsules
Existing formulations: Tablets, oral suspension
Indication: For the management of osteoarthritis pain
Zohydro ER (new formulation)
(Hydrocodone bitartrate; Zogenix)
Class/route: Opioid agonist; oral
New formulation: Extended-release capsules with BeadTek for an abuse deterrent
Existing formulation: Extended-release capsules
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
ACS indicates acute coronary syndrome; MI, myocardial infarction; QIDP, Qualified Infectious Disease Product; REMS, Risk Evaluation and Mitigation Strategy.
4. New Biosimilars, New Blood Products, New Vaccines, and New Viral Therapies
Bexsero (new vaccine)
(Meningococcal group B vaccine; Novartis)
Class/route: Vaccine; intramuscular injection
Indication: For active immunization to prevent and treat invasive disease caused by Neisseria meningitidis serogroup B in patients aged 10 years through 25 years
Approval considerations: Accelerated approval, breakthrough therapy
Fluad (new vaccine)
(Trivalent influenza vaccine; Novartis Vaccines and Diagnostics)
Class/route: Inactivated influenza vaccine; intramuscular injection
Indication: For active immunization against influenza disease caused by influenza virus subtypes A and B contained in the vaccine in patients aged ≥65 years
Approval consideration: Accelerated approval
Imlygic (new viral therapy)
(Talimogene laherparepvec; Amgen)
Class/route: First genetically modified oncolytic viral therapy; intralesional injection
Indication: For the local treatment of unresectable cutaneous, subcutaneous, and nodal lesions in patients with recurrent melanoma after initial surgery
Nuwiq (new blood product)
(Human coagulation factor VIII, simoctocog alfa; Octapharma USA)
Class/route: Recombinant factor VIII; intravenous injection
Indications: In adults and children with hemophilia A for on-demand treatment and control of bleeding episodes; for perioperative management of bleeding; and for routine prophylaxis to reduce the frequency of bleeding episodes
Zarxio (new biosimilar)
(filgrastim-sndz; Sandoz/Novartis)
Class/route: Leukocyte growth factor; subcutaneous, intravenous
New biosimilar: Biosimilar (of reference drug Neupogen): first-ever biosimilar approved in the United States
Indications: To decrease the incidence of infection, as manifested by febrile neutropenia, in patients with nonmyeloid malignancies receiving myelosuppressive anticancer drugs associated with a significant incidence of severe neutropenia with fever; for reducing the time to neutrophil recovery and the duration of fever after induction or consolidation chemotherapy in patients with acute myeloid leukemia; to reduce the duration of neutropenia and neutropenia-related clinical sequelae in patients with nonmyeloid malignancies undergoing myeloablative chemotherapy followed by bone marrow transplantation; for the mobilization of autologous hematopoietic progenitor cells into the peripheral blood for collection by leukapheresis; for long-term administration to reduce the incidence and duration of neutropenia sequelae in symptomatic patients with congenital neutropenia, cyclic neutropenia, or idiopathic neutropenia