FDA Approvals of Brand-Name Prescription Drugs in 2017

March 2018, Vol 11, Ninth Annual Payers' Guide - FDA Approvals, News & Updates
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The drugs included in this review were approved for the first time or received additional approvals by the US Food and Drug Administration in 2017 and are grouped here by several categories:

I. New Molecular Entities and New Biologic License Applications (NMEs and BLAs)
II. New Gene Therapies, Vaccines, and Blood Products
III. New Indications and New Combinations
IV. New Dosages, Dosage Forms, Formulations, Delivery Systems, and Patient Populations
V. New Biosimilars

 

I. New Molecular Entities and New Biologic License Applications

Aliqopa (NME)
(Copanlisib; Bayer HealthCare)
Class/route: Phosphatidylinositol-3-kinase inhibitor; intravenous injection
Indication: For treatment of adults with relapsed follicular lymphoma who received ≥2 systemic therapies
Approval considerations: Accelerated approval, fast track, priority review, orphan drug
Approval date: September 14, 2017

Alunbrig (NME)
(Brigatinib; Takeda Oncology)
Class/route: Tyrosine kinase inhibitor; oral tablets
Indication: For treatment of patients with ALK-positive metastatic NSCLC whose disease progressed with crizotinib or who are intolerant to crizotinib
Approval considerations: Accelerated approval, breakthrough therapy, priority review, orphan drug
Approval date: April 28, 2017

Austedo (NME)
(Deutetrabenazine; Teva)
Class/route: VMAT2 inhibitor; oral tablets
Indication: For treatment of patients with chorea associated with Huntington’s disease
Approval consideration: Orphan drug
Approval date: April 3, 2017
See also III. New Indications listing

Bavencio (BLA)
(Avelumab; EMD Serono/Pfizer)
Class/route: PD-L1–blocking antibody; intravenous injection
Indication: Treatment of all patients aged ≥12 years with metastatic Merkel-cell carcinoma, including those who have not received previous therapy.
Approval considerations: Accelerated approval, breakthrough therapy, fast track, priority review, orphan drug
Approval date: March 23, 2017
See also III. New Indications listing

Baxdela (NME)
(Delafloxacin; Melinta Therapeutics)
Class/routes: Fluoroquinolone antibacterial; oral tablets and intravenous injection
Indication: For treatment of adults with acute bacterial skin and skin structure infections caused by designated susceptible bacteria
Approval considerations: Fast track, priority review
Approval date: June 19, 2017

Benznidazole (NME)
(Benznidazole; Chemo Research)
(This drug was approved with no trade name)
Class/route: Nitroimidazole antimicrobial; oral tablets
Indication: For treatment of pediatric patients aged 2 to 12 years with Chagas disease (American trypanosomiasis) caused by Trypanosoma cruzi
Approval considerations: Accelerated approval, priority review, orphan drug
Approval date: August 29, 2017

Besponsa (BLA)
(Inotuzumab ozogamicin; Pfizer)
Class/route: First-in-class CD22-directed antibody-drug conjugate; intravenous injection
Indication: For treatment of adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia
Approval considerations: Breakthrough therapy, priority review, orphan drug
Approval date: August 17, 2017

Bevyxxa (NME)
(Betrixaban; Portola Pharmaceuticals)
Class/route: Factor Xa inhibitor; oral capsules
Indication: For prophylaxis of VTE in adults hospitalized for an acute illness who are at risk for thromboembolic complications caused by moderate or severe restricted mobility and other risk factors for VTE
Approval considerations: Fast track, priority review
Approval date: June 23, 2017

Brineura (BLA)
(Cerliponase alfa; BioMarin Pharmaceutical)
Class/route: First-in-class hydrolytic lysosomal N-terminal tripeptidyl peptidase; intraventricular injection
Indication: To slow the loss of ambulation in symptomatic pediatric patients aged ≥3 years with late infantile neuronal ceroid lipofuscinosis type 2 (or tripeptidyl peptidase 1 deficiency)
Approval considerations: Breakthrough therapy, priority review, orphan drug
Approval date: April 27, 2017

Calquence (NME)
(Acalabrutinib; AstraZeneca)
Class/route: Bruton’s tyrosine kinase inhibitor; oral capsules
Indication: For treatment of adults with mantle-cell lymphoma who received ≥1 therapies
Approval considerations: Accelerated approval, breakthrough therapy, priority review, orphan drug
Approval date: October 31, 2017

Dupixent (BLA)
(Dupilumab; Regeneron)
Class/route: First-in-class IL-4 receptor alpha antagonist; subcutaneous injection
Indication: For treatment, with or without topical corticosteroids, of adults with moderate-to-severe atopic dermatitis whose disease is not adequately controlled with topical prescription therapies, or when those therapies are not advisable
Approval considerations: Breakthrough therapy, priority review
Approval date: March 28, 2017

Emflaza (NME)
(Deflazacort; PTC Therapeutics)
Class/routes: First-in-class corticosteroid that reduces inflammation and immune system activity; oral tablets or oral suspension
Indication: For treatment of Duchenne muscular dystrophy in patients aged ≥5 years
Approval considerations: Fast track, priority review, orphan drug
Approval date: February 9, 2017

Fasenra (BLA)
(Benralizumab; AstraZeneca)
Class/route: IL-5 receptor alpha–directed cytolytic monoclonal antibody; subcutaneous injection
Indication: For add-on maintenance treatment of severe asthma in patients aged ≥12 years with an eosinophilic phenotype
Approval date: November 14, 2017

Giapreza (NME)
(Angiotensin II; La Jolla Pharmaceutical)
Class/route: First-in-class vasoconstrictor; intravenous injection
Indication: For treatment of hypotension in adults with distributive or vasodilatory shock
Approval consideration: Priority review
Approval date: December 21, 2017

Hemlibra (BLA)
(Emicizumab-kxwh; Genentech)
Class/route: First-in-class bispecific factor IXa– and factor X–directed antibody; subcutaneous injection
Indication: For routine prophylaxis to prevent or reduce bleeding episodes in adults and pediatric patients with hemophilia A and factor VIII inhibitors
Approval considerations: Breakthrough therapy, priority review, orphan drug
Approval date: November 16, 2017

Idhifa (NME)
(Enasidenib; Celgene)
Class/route: First-in-class IDH2 inhibitor; oral tablets
Indication: For treatment of adults with relapsed or refractory acute myeloid leukemia with IDH2 mutation, as detected by an FDA-approved test
Approval considerations: Fast track, priority review, orphan drug
Approval date: August 1, 2017

Imfinzi (BLA)
(Durvalumab; AstraZeneca)
Class/route: PD-L1–blocking antibody; intravenous injection
Indication: For treatment of patients with locally advanced or metastatic urothelial carcinoma whose
disease progressed during or after platinum-containing chemotherapy, or within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy
Approval considerations: Accelerated approval, breakthrough therapy, priority review
Approval date: May 1, 2017

Ingrezza (NME)
(Valbenazine; Neurocrine Biosciences)
Class/route: VMAT2 inhibitor; oral capsules
Indication: For treatment of adults with tardive dyskinesia
Approval considerations: Breakthrough therapy, fast track, priority review
Approval date: April 11, 2017

Kevzara (BLA)
(Sarilumab; Regeneron/Sanofi)
Class/route: IL-6 antagonist; subcutaneous injection
Indication: For treatment of adults with moderately to severely active rheumatoid arthritis who have had an inadequate response to or who are intolerant of ≥1 disease-modifying antirheumatic drugs
Approval date: May 22, 2017

Kisqali (NME)
(Ribociclib; Novartis)
Class/route: CDK4/CDK6 inhibitor; oral tablets
Indication: For treatment, in combination with an aromatase inhibitor as initial endocrine-based therapy, of postmenopausal women with HR-positive, HER2-­negative advanced or metastatic breast cancer
Approval considerations: Breakthrough therapy, priority review
Approval date: March 13, 2017

Kymriah See II. New Gene Therapies listing

Luxturna See II. New Gene Therapies listing

Macrilen (NME)
(Macimorelin; Aeterna Zentaris)
Class/route: First-in-class growth hormone secretagogue receptor (ghrelin) agonist; oral solution
Indication: For use in the diagnosis of adult growth hormone deficiency (diagnostic test)
Approval consideration: Orphan drug
Approval date: December 20, 2017

Mavyret (NME)
(Glecaprevir and pibrentasvir combination; AbbVie)
Classes/route: HCV NS3/4A protease inhibitor and HCV NS5A inhibitor; oral tablets
indications: For treatment of adults with chronic HCV genotype 1, 2, 3, 4, 5, or 6 infection without cirrhosis or with compensated cirrhosis (Child-Pugh A); and for treatment of adults with HCV genotype 1 infection who previously received a regimen containing an HCV NS5A or NS3/4A protease inhibitor, but not both
Approval considerations: Fast track, breakthrough therapy, priority review
Approval date: August 3, 2017

Mepsevii (BLA)
(Vestronidase alfa-vjbk; Ultragenyx Pharmaceutical)
Class/route: First-in-class recombinant human lysosomal beta glucuronidase; intravenous injection
Indication: For treatment of mucopolysaccharidosis type VII (Sly syndrome) in pediatric patients and adults
Approval considerations: Fast track, priority review, orphan drug
Approval date: November 15, 2017

Nerlynx (NME)
(Neratinib; Puma)
Class/route: Kinase inhibitor; oral tablets
Indication: For extended adjuvant treatment of adults with early-stage HER2-overexpressed/amplified breast cancer, after adjuvant trastuzumab-based therapy
Approval date: July 17, 2017

Ocrevus (BLA)
(Ocrelizumab; Genentech)
Class/route: First-in-class CD20-directed cytolytic antibody; intravenous injection
Indication: For treatment of patients with relapsing or primary progressive forms of multiple sclerosis
Approval considerations: Breakthrough therapy, fast track, priority review
Approval date: March 28, 2017

Ozempic (NME)
(Semaglutide; Novo Nordisk)
Class/route: Glucagon-like peptide 1 receptor agonist; subcutaneous injection
Indication: To improve glycemic control in adults with type 2 diabetes mellitus, as an adjunct to diet and exercise
Approval date: December 5, 2017

Parsabiv (NME)
(Etelcalcetide; Amgen)
Class/route: Calcimimetic; intravenous injection
Indication: For treatment of secondary hyperparathyroidism in adults with chronic kidney disease undergoing hemodialysis
Approval date: February 7, 2017

Prevymis (NME)
(Letermovir; Merck)
Class/routes: First-in-class CMV DNA terminase complex inhibitor; oral tablets and intravenous injection
Indication: For prophylaxis of CMV infection and disease in adult CMV-seropositive recipients of an allogeneic hematopoietic stem-cell transplant
Approval considerations: Breakthrough therapy, fast track, priority review, orphan drug
Approval date: November 8, 2017

Radicava (NME)
(Edaravone; Mitsubishi Tanabe)
Class/route: First-in-class substituted 2-pyrazolin-5-one; intravenous injection
Indication: For treatment of patients with amyotrophic lateral sclerosis (Lou Gehrig disease)
Approval consideration: Orphan drug
Approval date: May 5, 2017

Rhopressa (NME)
(Netarsudil; Aerie Pharmaceuticals)
Class/route: First-in-class Rho kinase inhibitor; topical ophthalmic solution
Indication: For reduction of elevated intraocular pressure in patients with open-angle glaucoma or with ocular hypertension
Approval date: December 18, 2017

Rydapt (NME)
(Midostaurin; Novartis)
Class/route: First-in-class kinase inhibitor; oral capsules
Indication: For treatment, in combination with standard cytarabine and daunorubicin induction and cytarabine consolidation, of adults with newly diagnosed acute myeloid leukemia and FLT3 mutation, as detected by an FDA-approved test
Approval considerations: Breakthrough therapy, fast track, priority review, orphan drug
Approval date: April 28, 2017

Siliq (BLA)
(Brodalumab; Valeant)
Class/route: Human IL-17 receptor A antagonist; sub­cutaneous injection
Indication: For treatment of moderate-to-severe plaque psoriasis in adults who are candidates for systemic therapy or for phototherapy and who were unresponsive or lost response to other systemic therapies
Approval consideration: REMS program
Approval date: February 15, 2017

Solosec (NME)
(Secnidazole; Symbiomix Therapeutics)
Class/route: Nitroimidazole antimicrobial; oral granules
Indication: For treatment of bacterial vaginosis in adult women
Approval considerations: Fast track, priority review
Approval date: September 15, 2017

Steglatro (NME)
(Ertugliflozin; Merck)
Class/route: Sodium glucose cotransporter 2 inhibitor; oral tablets
Indication: To improve glycemic control in adults with type 2 diabetes mellitus, as an adjunct to diet and exercise
Approval date: December 19, 2017

Symproic (NME)
(Naldemedine; Purdue Pharma)
Class/route: Opioid antagonist; oral tablets
Indication: For treatment of opioid-induced constipation in adults with chronic noncancer pain, including chronic pain related to previous cancer or its treatment in patients who do not require frequent opioid dosage escalation
Approval date: March 23, 2017

Tremfya (BLA)
(Guselkumab; Janssen Biotech)
Class/route: IL-23 blocker; subcutaneous injection
Indication: For treatment of adults with moderate-to-­severe plaque psoriasis who are candidates for systemic therapy or for phototherapy
Approval date: July 13, 2017

Trulance (NME)
(Plecanatide; Synergy Pharmaceuticals)
Class/route: Guanylate cyclase-C agonist; oral tablets
Indication: For treatment of chronic idiopathic constipation in adults
Approval date: January 19, 2017

Tymlos (NME)
(Abaloparatide; Radius Health)
Class/route: Human parathyroid hormone–related peptide (1-34) analog; subcutaneous injection
Indication: For treatment of postmenopausal women with osteoporosis at high risk for fracture or patients who did not respond to or are intolerant of other available osteo­porosis therapies
Approval date: April 28, 2017

Vabomere (NME)
(Meropenem and vaborbactam combination; The Medicines Company)
Classes/route: Penem antibacterial and beta-lactamase inhibitor; intravenous injection
Indication: For treatment of complicated urinary tract infections, including pyelonephritis caused by Escherichia coli, Klebsiella pneumoniae, and Enterobacter cloacae species complex, in patients aged ≥18 years
Approval considerations: Fast track, priority review
Approval date: August 29, 2017

Verzenio (NME)
(Abemaciclib; Eli Lilly)
Class/route: CDK4/CDK6 inhibitor; oral tablets
indications: For treatment, in combination with fulvestrant, of women with HR-positive, HER2-negative advanced or metastatic breast cancer whose disease progressed after endocrine therapy; and as monotherapy for treatment of adults with HR-positive, HER2-negative advanced or metastatic breast cancer whose disease progressed after endocrine therapy and chemotherapy in the metastatic setting
Approval considerations: Breakthrough therapy, fast track, priority review
Approval date: September 28, 2017
[New indication February 26, 2018]

Vosevi (NME)
(Sofosbuvir, velpatasvir, and voxilaprevir combination; Gilead Sciences)
Classes/route: HCV nucleotide analog NS5B polymerase inhibitor, HCV NS5A inhibitor, and HCV NS3/4A protease inhibitor; oral tablets
Indication: For treatment of adults with chronic HCV infection without cirrhosis or with compensated cirrhosis (Child-Pugh A) who have genotype 1, 2, 3, 4, 5, or 6 infection and have previously received an HCV regimen containing an NS5A inhibitor, or who have genotype 1a or 3 infection and have previously received an HCV regimen containing sofosbuvir without an NS5A inhibitor
Approval considerations: Breakthrough therapy, fast track, priority review
Approval date: July 18, 2017

Vyzulta (NME)
(Latanoprostene bunod; Bausch & Lomb)
Class/route: Prostaglandin analog; topical ophthalmic solution
Indication: To reduce intraocular pressure in patients with open-angle glaucoma or with ocular hypertension
Approval date: November 2, 2017

Xadago (NME)
(Safinamide; Newron Pharmaceuticals/US WorldMeds)
Class/route: Monoamine oxidase type B inhibitor; oral tablets
Indication: As adjunctive treatment to levodopa and carbidopa in patients with Parkinson’s disease who are experiencing “off” episodes
Approval date: March 21, 2017

Xepi (NME)
(Ozenoxacin; Medimetriks)
Class/route: Quinolone antimicrobial; topical cream
Indication: For topical treatment of impetigo caused by Staphylococcus aureus or Streptococcus pyogenes in patients aged ≥2 months
Approval date: December 11, 2017

Xermelo (NME)
(Telotristat ethyl; Lexicon)
Class/route: First-in-class tryptophan hydroxylase inhibitor; oral tablets
Indication: For treatment of carcinoid syndrome diarrhea, in combination with SSA therapy, in adults whose condition is inadequately controlled by SSA therapy
Approval considerations: Fast track, priority review, orphan drug
Approval date: February 28, 2017

Yescarta See II. New Gene Therapies listing

Zejula (NME)
(Niraparib; Tesaro)
Class/route: Poly(ADP-ribose) polymerase inhibitor
Indication: For maintenance treatment of adult women with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who have a complete or partial response to platinum-based chemotherapy
Approval considerations: Breakthrough therapy, fast track, priority review, orphan drug
Approval date: March 27, 2017

ALK indicates anaplastic lymphoma kinase; BLA, biologic license application; CDK, cyclin-dependent kinase; CMV, cytomegalovirus; FDA, US Food and Drug Administration; HCV, hepatitis C virus; HER2, human epidermal growth factor receptor 2; HR, hormone receptor; IDH2, isocitrate dehydrogenase-2; IL, interleukin; NME, new molecular entity; NSCLC, non–small-cell lung cancer; PD-L1, programmed-cell death ligand 1; REMS, Risk Evaluation and Mitigation Strategy; SSA, somatostatin analog; VMAT2, vesicular monoamine transporter; VTE, venous thromboembolism.

 

II. New Gene Therapies, Vaccines, and Blood Products

Haegarda (new blood product)
(C1 esterase inhibitor [human]; CSL Behring)
Class/route: First-in-class plasma-derived C1 esterase inhibitor (human); freeze-dried powder for reconstitution for subcutaneous injection
Indication: For routine prophylaxis to prevent hereditary angioedema attacks in adolescents and adults
Approval date: June 22, 2017

Heplisav-B (new vaccine)
(Hepatitis B vaccine recombinant, adjuvanted; Dynavax)
Class/route: Vaccine; intramuscular injection
Indication: For prevention of infection caused by all known subtypes of hepatitis B virus in adults aged ≥18 years
Approval date: November 9, 2017

Kymriah (new gene therapy)
(Tisagenlecleucel; Novartis)
Class/route: First-in-class CD19-directed genetically modified autologous (CAR) T-cell immunotherapy; intravenous injection
Indication: For treatment of young patients aged ≤25 years with B-cell precursor acute lymphoblastic leukemia that is refractory to other treatments or is in second or later relapse
Approval considerations: Breakthrough therapy, priority review; REMS
Approval date: August 30, 2017

Luxturna (new gene therapy)
(Voretigene neparvovec-rzyl; Spark Therapeutics)
Class/route: First-in-class adeno-associated virus vector-based gene therapy; intraocular suspension for subretinal injection
Indication: For treatment of patients with confirmed biallelic RPE65 mutation–associated retinal dystrophy that leads to vision loss
Approval considerations: Breakthrough therapy, orphan drug, priority review
Approval date: December 19, 2017

Rebinyn (new blood product)
(Coagulation factor IX recombinant, glycopegylated; Novo Nordisk)
Class/route: Recombinant DNA-derived coagulation factor IX concentrate; lyophilized powder for solution for intravenous injection
indications: For on-demand treatment and control of bleeding episodes, and for perioperative management of bleeding, in adults and children with hemophilia B
Approval date: May 31, 2017

Shingrix (new vaccine)
(Zoster vaccine recombinant, adjuvanted; GlaxoSmithKline)
Class/route: Vaccine; intramuscular suspension
Indication: For prevention of herpes zoster infection in adults aged ≥50 years
Approval date: October 20, 2017

Yescarta (new gene therapy)
(Axicabtagene ciloleucel; Kite Pharma)
Class/route: CD19-directed genetically modified autologous (CAR) T-cell immunotherapy; intravenous injection
Indication: For treatment of adults with relapsed or refractory large B-cell lymphoma after ≥2 lines of systemic therapies, including DLBCL not otherwise specified, primary mediastinal large B-cell lymphoma, high-grade B-cell lymphoma, and DLBCL arising from follicular lymphoma
Approval considerations: Breakthrough therapy, priority review, orphan drug; REMS
Approval date: October 18, 2017

CAR indicates chimeric antigen receptor; DLBCL, diffuse large B-cell lymphoma; REMS, Risk Evaluation and
Mitigation Strategy.

 

III. New Indications and New Combinations

Abilify MyCite (new combination)
(Aripiprazole with sensor; Otsuka Pharmaceutical)
Class/route: Atypical antipsychotic and ingestible sensor; oral tablets with sensor
New combination: A drug-sensor combination comprised of aripiprazole tablets embedded with an ingestible event marker sensor intended to track drug ingestion
indications: For treatment of adults with schizophrenia; treatment of bipolar I disorder; treatment of adults with acute manic and mixed episodes as monotherapy and as adjunct to lithium or valproate therapy, or as maintenance treatment of adults as monotherapy and as adjunct to lithium or valproate therapy; adjunctive treatment of adults with major depressive disorder
Approval date: November 13, 2017

Actemra (new indications)
(Tocilizumab; Genentech)
Class/route: IL-6 receptor antagonist; intravenous or subcutaneous injection
New indications: First drug for adults with giant-cell arteritis; first treatment for CAR T-cell–induced severe or life-threatening cytokine release syndrome in patients aged ≥2 years
Existing indications: For treatment of adults with moderately to severely active rheumatoid arthritis; treatment of polyarticular juvenile idiopathic arthritis and systemic juvenile idiopathic arthritis in patients aged ≥2 years
Approval considerations: Breakthrough therapy (giant-­cell arteritis), orphan drug (CAR T-cell–induced cytokine release syndrome and giant-cell arteritis), priority review (giant-cell arteritis)
Approval dates: May 22, 2017 (giant-cell arteritis); August 30, 2017 (cytokine release syndrome)

Alecensa (new indication)
(Alectinib; Genentech)
Class/route: Tyrosine kinase inhibitor; oral capsules
New Indication: For first-line treatment of patients with ALK-positive metastatic NSCLC, as detected by an FDA-approved test
Existing Indication: For treatment of patients with ALK-positive metastatic NSCLC whose disease progressed during or who were intolerant of crizotinib therapy
Approval consideration: Breakthrough therapy
Approval date: November 6, 2017

Austedo (new indication)
(Deutetrabenazine; Teva)
Class/route: VMAT2 inhibitor; oral tablets
Indication: For treatment of tardive dyskinesia in adults
Existing Indication: For treatment of chorea associated with Huntington’s disease
Approval date: August 30, 2017
See also I. NMEs and BLAs listing

Bavencio (new indication)
(Avelumab; EMD Serono/Pfizer)
Class/route: PD-L1–blocking monoclonal antibody; intravenous injection
New Indication: For treatment of patients with locally advanced or metastatic urothelial carcinoma whose disease progressed during or after platinum-containing chemotherapy, or whose disease progressed within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy
Existing Indication: Treatment of patients aged ≥12 years with metastatic Merkel-cell carcinoma
Approval consideration: Accelerated approval
Approval date: May 9, 2017
See also I. NMEs and BLAs listing

Blincyto (new indication)
(Blinatumomab; Amgen)
Class/route: Bispecific CD19-directed CD3 T-cell engager; intravenous injection
New Indication: For treatment of Ph+ relapsed or refractory B-cell precursor acute lymphoblastic leukemia in adults and children
Existing Indication: For treatment of Ph– relapsed or refractory B-cell precursor acute lymphoblastic leukemia
Approval date: July 11, 2017

Bosulif (new indication)
(Bosutinib; Pfizer)
Class/route: Tyrosine kinase inhibitor (BCR-ABL inhibitor); oral tablets
New Indication: For treatment of newly diagnosed chronic-phase Ph+ chronic myelogenous leukemia
Existing indications: For treatment of chronic-, accelerated-, or blast-phase Ph+ chronic myelogenous leukemia that is resistant to or intolerant of previous therapy
Approval considerations: Accelerated approval, orphan drug, priority review
Approval date: December 19, 2017

Cabometyx (new indication)
(Cabozantinib; Exelixis)
Class/route: Tyrosine kinase inhibitor; oral tablets
New Indication: For first-line treatment of patients with advanced renal-cell carcinoma
Existing Indication: For treatment of patients with advanced renal-cell carcinoma who have received antiangiogenic therapy
Approval consideration: Priority review
Approval date: December 19, 2017

Darzalex (new indication)
(Daratumumab; Janssen Biotech)
Class/route: CD38-directed cytolytic antibody; intravenous injection
New Indication: For treatment, in combination with pomalidomide and dexamethasone, of patients with multiple myeloma who have received ≥2 therapies, including lenalidomide and a proteasome inhibitor
Existing indications: For treatment, in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, of patients with multiple myeloma who have received ≥1 therapies; as monotherapy for treatment of patients with multiple myeloma who have received ≥3 lines of therapy, including a proteasome inhibitor and an immunomodulatory agent, or whose disease is double refractory to a proteasome inhibitor and an immunomodulatory agent
Approval date: June 16, 2017

Duzallo (new combination)
(Lesinurad and allopurinol; Ironwood Pharmaceuticals)
Class/route: URAT1 inhibitor and xanthine oxidase inhibitor; oral tablets
Indication: For treatment of hyperuricemia associated with gout in patients who have not achieved target serum uric acid levels with an appropriate daily dose of allopurinol alone
Approval date: August 18, 2017

Dysport (new indication)
(AbobotulinumtoxinA; Ipsen)
Classes/route: Acetylcholine release inhibitor and a neuromuscular-blocking agent; intramuscular injection
New Indication: For treatment of adults with lower-limb spasticity
Existing indications: For treatment of adults with cervical dystonia; for temporary improvement in the appearance of moderate-to-severe glabellar lines associated with procerus and corrugator muscle activity in patients aged <65 years; treatment of upper-limb spasticity in adults; treatment of lower-limb spasticity in pediatric patients aged ≥2 years
Approval date: June 14, 2017

Gazyva (new indication)
(Obinutuzumab; Genentech)
Class/route: CD20-directed cytolytic antibody; intravenous injection
New Indication: For first-line treatment, in combination with chemotherapy, followed by obinutuzumab monotherapy in patients achieving at least partial remission, of patients with untreated stage II bulky, stage III, or stage IV follicular lymphoma in adults
Existing indications: For treatment, in combination with chlorambucil, of untreated chronic lymphocytic leukemia; treatment, in combination with bendamustine followed by obinutuzumab monotherapy, of follicular lymphoma in patients whose disease relapsed after or is refractory to a rituximab-containing regimen
Approval consideration: Priority review
Approval date: November 16, 2017

Ibrance (new indication)
(Palbociclib; Pfizer)
Class/route: CDK4/CDK6 inhibitor; oral capsules
New Indication: For treatment, in combination with an aromatase inhibitor as initial endocrine-based therapy, of postmenopausal women with HR-positive, HER2-­negative advanced or metastatic breast cancer
Existing indications: For treatment, in combination with letrozole, of ER-positive, HER2-negative advanced breast cancer as initial endocrine-based therapy in postmenopausal women; treatment, in combination with fulvestrant, of HR-positive, HER2-negative advanced or metastatic breast cancer in women whose disease progressed after endocrine therapy
Approval considerations: Breakthrough therapy, priority review
Approval date: March 31, 2017

Imbruvica (new indications)
(Ibrutinib; Pharmacyclics)
Class/route: Bruton’s tyrosine kinase inhibitor; oral capsules/tablets
New indications: First treatment for relapsed or refractory marginal-zone lymphoma (MZL) in patients requiring systemic therapy, ≥1 anti-CD20–based therapies; first treatment for adults with chronic graft-versus-host disease (GVHD), after failure of ≥1 systemic therapies
Existing indications: For treatment of chronic lympho­cytic leukemia or small lymphocytic lymphoma, with
or without 17p deletion; treatment of Waldenström’s macroglobulinemia; treatment of mantle-cell lymphoma
Approval considerations: Accelerated approval (MZL), breakthrough therapy (GVHD, MZL), orphan drug (both), priority review (GVHD)
Approval dates: January 19, 2017 (MZL); August 2, 2017 (GVHD)
[New dose February 16, 2018: once-daily tablets]

Juluca (new combination)
(Dolutegravir and rilpivirine; ViiV Healthcare)
Class/route: HIV-1 integrase strand transfer inhibitor and nonnucleoside reverse transcriptase inhibitor; oral tablets
Indication: First 2-drug complete regimen for treatment of HIV type 1 (HIV-1) infection in adults to replace the current antiretroviral regimen with a stable antiretroviral regimen for ≥6 months in patients with virologically suppressed disease (HIV-1 RNA <50 copies/mL) and no history of treatment failure and no known substitutions associated with resistance to the individual components of Juluca
Approval consideration: Priority review
Approval date: November 21, 2017

Kalydeco (new indication)
(Ivacaftor; Vertex Pharmaceuticals)
Class/route: CFTR potentiator; oral tablets
New Indication: For treatment of cystic fibrosis associated with 1 of 33 CFTR gene mutations
Existing Indication: For treatment of patients aged ≥2 years with cystic fibrosis associated with 1 of 10 mutations in the CFTR gene that is responsive to ivacaftor based on clinical and/or in vitro assay data
Approval date: May 17, 2017
See also IV. New Patient Population listing

Keytruda (new indications)
(Pembrolizumab; Merck)
Class/route: PD-1–blocking antibody; intravenous injection
New indications: For treatment of refractory classical Hodgkin lymphoma or disease that relapsed after ≥3 previous therapies; treatment, in combination with pemetrexed and carboplatin, of previously untreated metastatic nonsquamous NSCLC; treatment of locally advanced or metastatic urothelial carcinoma in patients who are not eligible for cisplatin-containing chemotherapy; treatment of locally advanced or metastatic urothelial carcinoma in patients whose disease progressed during or after platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy; first drug approved based on a biomarker for treatment of unresectable or metastatic MSI-H or dMMR solid tumor that progressed after previous treatment, and for MSI-H or dMMR colorectal cancer that progressed after treatment with fluoropyrimidine, oxaliplatin, and irinotecan; treatment of recurrent, locally advanced or metastatic gastric or gastroesophageal junction adenocarcinoma in patients whose tumors express PD-L1
Existing indications: For treatment of unresectable or metastatic melanoma; for first-line monotherapy of metastatic NSCLC in patients whose tumors have high PD-L1 expression; treatment of recurrent or metastatic head and neck squamous-cell cancer in patients whose disease progressed during or after platinum-containing chemotherapy
Approval considerations: Accelerated approval (NSCLC, first-line for urothelial carcinoma, solid tumors with MSI-H or dMMR biomarker); breakthrough therapy (second-line for urothelial carcinoma); priority review (NSCLC, urothelial carcinoma, solid tumors with MSI-H or dMMR biomarkers)
Approval dates: March 15, 2017 (classical Hodgkin lymphoma); May 10, 2017 (NSCLC); May 18, 2017 (urothelial cancer); May 23, 2017 (solid tumor with MSI-H or dMMR biomarker); September 22, 2017 (gastric cancer)

Lucentis (new indications)
(Ranibizumab; Genentech)
Class/route: VEGF inhibitor; intravitreal injection
New indications: For treatment of myopic choroidal neovascularization; treatment of diabetic retinopathy in patients with diabetic macular edema
Existing indications: Treatment of neovascular (wet) age-related macular degeneration; treatment of macular edema after retinal vein occlusion; treatment of diabetic macular edema
Approval considerations: Breakthrough therapy (diabetic retinopathy); priority review (diabetic retinopathy, myopic choroidal neovascularization)
Approval dates: January 5, 2017 (myopic choroidal neovascularization); April 17, 2017 (diabetic retinopathy)

Lynparza (new indication)
(Olaparib; AstraZeneca)
Class/route: PARP inhibitor; oral tablets, oral capsules
New Indication: For maintenance treatment of recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer in adults who have had a complete or partial response to platinum-based chemotherapy
Existing indications: For treatment of advanced ovarian cancer in patients with deleterious or suspected deleterious germline BRCA mutation who have received ≥3 lines of chemotherapy; treatment of advanced ovarian cancer in patients with deleterious or suspected deleterious germline BRCA mutation who have received ≥3 lines of chemotherapy
Approval consideration: Fast track
Approval date: August 17, 2017
[New indication January 12, 2018: For treatment of HER2-negative metastatic breast cancer in patients with deleterious or suspected deleterious germline BRCA mutation who have received chemotherapy]
See also IV. New Dosage Form listing

Mavyret (new combination) See I. NMEs and BLAs listing

Mylotarg (new indications)
(Gemtuzumab ozogamicin; Pfizer)
New indications: For treatment of newly diagnosed CD33-positive acute myeloid leukemia in adults; treatment of relapsed or refractory CD33-positive acute myeloid leukemia in pediatric patients aged ≥2 years
Existing Indication: Initially approved (in 2000) at a higher dose for treatment of adults with relapsed CD33-positive acute myeloid leukemia, this drug was withdrawn from the market (in 2010) by the manufacturer
Approval consideration: Orphan drug
Approval date: September 1, 2017
See also IV. New Dosage/Patient Population listing

Nucala (new indication)
(Mepolizumab; GlaxoSmithKline)
Class/route: IL-5 antagonist monoclonal antibody; subcutaneous injection
New Indication: For treatment of adults with eosinophilic granulomatosis and polyangiitis
Existing Indication: Add-on maintenance treatment of patients with severe asthma aged ≥12 years with an eosinophilic phenotype
Approval considerations: Orphan drug, priority review
Approval date: December 12, 2017

Opdivo (new indications)
(Nivolumab; Bristol-Myers Squibb)
Class/route: PD-1–blocking antibody; intravenous injection
New indications: For adjuvant treatment of patients with melanoma with lymph node involvement or in patients with metastatic disease who had complete resection; treatment of hepatocellular carcinoma in patients who have previously received sorafenib; treatment of locally advanced or metastatic urothelial carcinoma; treatment of patients aged ≥12 years with MSI-H or dMMR metastatic colorectal cancer that progressed after treatment with fluoropyrimidine, oxaliplatin, and irinotecan
Existing indications: For treatment, in combination with ipilimumab, of patients with BRAF V600 wild-type and BRAF V600 mutation–positive unresectable or metastatic melanoma; treatment of classical Hodgkin lymphoma that has relapsed or progressed after autologous hematopoietic stem-cell transplantation and posttransplant brentuximab vedotin; treatment of recurrent or metastatic squamous-cell carcinoma of the head and neck; treatment of metastatic NSCLC; treatment of renal-cell carcinoma
Approval considerations: Accelerated approval (hepatocellular carcinoma, colorectal cancer); breakthrough therapy (melanoma, urothelial carcinoma); priority review (colorectal cancer, hepatocellular carcinoma, melanoma, urothelial carcinoma)
Approval dates: February 2, 2017 (urothelial carcinoma); July 31, 2017 (colorectal cancer); September 22, 2017 (hepatocellular carcinoma); December 20, 2017 (melanoma)

Qtern (new combination)
(Dapagliflozin and saxagliptin; AstraZeneca)
Class/route: SGLT-2 inhibitor and DPP-4 inhibitor; oral tablets
Indication: To improve glycemic control, as an adjunct to diet and exercise, in adults with type 2 diabetes mellitus and inadequate glycemic control with dapagliflozin or who are already receiving dapagliflozin and saxagliptin
Approval date: February 27, 2017

Revlimid (new indication)
(Lenalidomide; Celgene)
Class/route: Thalidomide analog; oral capsules
New Indication: First drug for maintenance therapy in patients with multiple myeloma after autologous stem-cell transplant
Existing Indication: For treatment, in combination with dexamethasone therapy, of multiple myeloma; for treatment of transfusion-dependent anemia that results from low- or intermediate-1–risk myelodysplastic syndromes associated with a deletion 5q abnormality, with or without additional cytogenetic abnormalities; treatment of mantle-cell lymphoma in patients whose disease has relapsed or progressed after 2 therapies, one of which included bortezomib
Approval consideration: REMS
Approval date: February 22, 2017

Rituxan Hycela (new combination)
(Rituximab and hyaluronidase human; Genentech)
Class/route: CD20-directed cytolytic antibody and endoglycosidase; subcutaneous injection
indications: For treatment of adults with untreated relapsed or refractory follicular lymphoma; treatment of untreated diffuse large B-cell lymphoma, in combination with cyclophosphamide, doxorubicin, vincristine, and prednisone, or with other anthracycline-based chemotherapy regimens; treatment, in combination with fludarabine and cyclophosphamide, of patients with untreated or treated chronic lymphocytic leukemia
Approval date: June 22, 2017

Simponi Aria (new indications)
(Golimumab; Janssen Biotech)
Class/route: Fully human anti–tumor necrosis factor-­alpha monoclonal antibody; intravenous injection
New indications: For treatment of adults with active psoriatic arthritis; treatment of active ankylosing spondylitis
Existing Indication: For treatment, in combination with methotrexate, of adults with moderately to severely active rheumatoid arthritis
Approval date: October 20, 2017

Soliris (new indication)
(Eculizumab; Alexion)
Class/route: Complement inhibitor; intravenous injection
New Indication: For treatment of generalized myasthenia gravis in adults with anti–acetylcholine receptor antibody
Existing indications: Treatment of patients with parox­ysmal nocturnal hemoglobinuria to reduce hemolysis; treatment of atypical hemolytic uremic syndrome to inhibit complement-mediated thrombotic microangiopathy
Approval consideration: Orphan drug, REMS
Approval date: October 23, 2017

Somatuline Depot (new indications)
(Lanreotide; Ipsen)
Class/route: Somatostatin analog; subcutaneous injection
New indications: For treatment of adults with carcinoid syndrome; to reduce the frequency of short-acting somatostatin analog rescue therapy
Existing indications: For long-term treatment of patients with acromegaly who have had an inadequate response to or cannot have surgery and/or radiotherapy; treatment of adults with unresectable, well- or moderately differentiated, locally advanced or metastatic gastroenteropancreatic neuroendocrine tumors to improve progression-free survival
Approval date: September 18, 2017

Stivarga (new indication)
(Regorafenib; Bayer HealthCare)
Class/route: Tyrosine kinase inhibitor; oral tablets
New Indication: For treatment of hepatocellular carcinoma in patients who have received sorafenib
Existing indications: For treatment of metastatic colorectal cancer in patients who have received fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy, an anti-VEGF therapy, and, if RAS wild-type, an anti-­EGFR therapy; for treatment of locally advanced, unresectable or metastatic GIST in patients who have received imatinib mesylate and sunitinib malate therapy
Approval considerations: Orphan drug, priority review
Approval date: April 27, 2017

Sutent (new indication)
(Sunitinib malate; Pfizer)
Class/route: Tyrosine kinase inhibitor; oral capsules
New Indication: For adjuvant treatment of adults at high risk for recurrent renal-cell carcinoma after nephrectomy
Existing indications: Treatment of GIST in patients who are intolerant of or whose disease progressed after imatinib mesylate; treatment of advanced renal-cell carcinoma; treatment of progressive, well-differentiated unresectable locally advanced or metastatic pancreatic neuroendocrine tumors
Approval date: November 16, 2017

Tafinlar and Mekinist (new indication)
(DaBRAFenib and trametinib; Novartis)
Class/routes: Kinase inhibitor of BRAF pathway: kinase inhibitor of MEK1 and MEK2 pathway; oral capsules and oral tablets
New Indication: For treatment of metastatic NSCLC with BRAF V600E mutation, as detected by an FDA-approved test
Existing Indication: For treatment of unresectable or metastatic melanoma in patients with BRAF V600E or V600K mutations, as detected by an FDA-approved test
Approval date: June 22, 2017

Tasigna (new indication)
(Nilotinib; Novartis)
Class/route: BCR-ABL kinase inhibitor; oral capsules
New Indication: First FDA approval for treatment discontinuation of a drug in patients with chronic-phase chronic myeloid leukemia who have been taking nilotinib for ≥3 years and whose disease has responded to treatment, as detected by an FDA-approved test
Existing indications: Treatment (ongoing) of adults with newly diagnosed chronic-phase Ph+ chronic myeloid leukemia; treatment (ongoing) of chronic-phase and accelerated-phase Ph+ chronic myeloid leukemia in adults with disease that is resistant to or who are intolerant of previous therapy that included imatinib
Approval considerations: Orphan drug, priority review
Approval date: December 22, 2017

Tecentriq (new indication)
(Atezolizumab; Genentech)
Class/route: PD-L1–blocking antibody; intravenous injection
New Indication: For first-line treatment of patients with locally advanced or metastatic urothelial carcinoma who are not eligible for cisplatin-containing chemotherapy
Existing indications: Treatment of locally advanced or metastatic urothelial carcinoma in patients whose disease progressed during or after a platinum-containing chemotherapy, or within 12 months of neoadjuvant or adjuvant chemotherapy; treatment of metastatic NSCLC in patients whose disease progressed during or after platinum-containing chemotherapy
Approval consideration: Accelerated approval
Approval date: April 17, 2017

Vabomere (new combination) See I. NMEs and BLAs listing

Victoza (new indication)
(Liraglutide; Novo Nordisk)
Class/route: GLP-1 receptor agonist; subcutaneous injection
New Indication: To reduce the risk for major adverse cardiovascular events in adults with type 2 diabetes and established cardiovascular disease
Existing Indication: To improve glycemic control in adults with type 2 diabetes, as an adjunct to diet and exercise
Approval date: August 25, 2017

Vosevi (new combination) See I. NMEs and BLAs listing

Vyxeos (new combination)
(Daunorubicin and cytarabine; Jazz Pharmaceuticals)
Classes/route: Anthracycline topoisomerase inhibitor and nucleoside metabolic inhibitor; intravenous injection
indications: For treatment of adults with newly diagnosed therapy-related acute myeloid leukemia; treatment of adults with newly diagnosed acute myeloid leukemia with myelodysplasia-related changes
Approval considerations: Breakthrough therapy, orphan drug, priority review
Approval date: August 3, 2017

Xeljanz/Xeljanz XR (new indication)
(Tofacitinib; Pfizer)
Class/route: Janus kinase inhibitor; oral tablets
New Indication: For treatment of adults with active psoriatic arthritis who have had an inadequate response to or are intolerant of methotrexate or other disease-modifying antirheumatic drugs
Existing Indication: For treatment of adults with moderately to severely active rheumatoid arthritis who have had an inadequate response to or are intolerant of methotrexate
Approval date: December 14, 2017

Zelboraf (new indication)
(Vemurafenib; Genentech)
Class/route: BRAF serine-threonine kinase inhibitor; oral tablets
New Indication: First treatment for patients with Erdheim-Chester Disease with BRAF V600 mutation
Existing indications: Treatment of patients with unresectable or metastatic melanoma with BRAF V600E mutation, as detected by an FDA-approved test; treatment, in combination with cobimetinib, of patients with unresectable or metastatic melanoma with BRAF V600E or V600K mutation
Approval considerations: Breakthrough therapy, orphan drug, priority review
Approval date: November 6, 2017

Zykadia (new indication)
(Ceritinib; Novartis)
Class/route: Tyrosine kinase inhibitor; oral capsules
New Indication: For treatment of metastatic NSCLC with ALK mutation, as detected by an FDA-approved test
Existing indications: For treatment of metastatic NSCLC with ALK mutation in patients whose disease has progressed with or who are intolerant to crizotinib
Approval consideration: Priority review
Approval date: May 26, 2017

ALK indicates anaplastic lymphoma kinase; CAR, chimeric antigen receptor; CDK, cyclin-dependent kinase; dMMR, mismatch repair–deficient biomarker; DPP-4, dipeptidyl peptidase-4; EGFR, epidermal growth factor receptor; FDA, US Food and Drug Administration; GIST, gastrointestinal stromal tumor; GLP-1, glucagon-like peptide-1; HER, human epidermal growth factor receptor; HR, hormone receptor; IL, interleukin; MEK, mitogen-activated extracellular signal-regulated kinase; MSI-H, microsatellite instability-high; NSCLC, non–small-cell lung cancer; PARP, (poly ADP-ribose) polymerase; PD-1/PD-L1, programmed-cell death receptor 1/ligand 1; Ph–, Philadelphia chromosome–negative; Ph+, Philadelphia chromosome–positive; REMS, Risk Evaluation and Mitigation Strategy; SGLT-2, sodium-glucose cotransporter 2; VEGF, vascular endothelial growth factor.

IV. New Dosages, Dosage Forms, Formulations, and Patient Populations

Admelog (new formulation)
(Insulin lispro injection; sanofi aventis)
Class/route: Rapid-acting human insulin analog; subcutaneous or intravenous
New formulation: First short-acting insulin approved as follow-on therapy
indications: To improve glycemic control in adults and pediatric patients aged ≥3 years with type 1 diabetes, and in adults with type 2 diabetes
Approval date: December 11, 2017

Arymo ER (new formulation)
(Morphine sulfate extended release; Egalet)
Class/route: Opioid agonist; oral tablets
New formulation: 15-mg, 30-mg, and 60-mg film-coated, extended-release tablets with an abuse-deterrent formulation
Indication: For management of pain severe enough to require daily, around-the-clock, long-term opioid treatment in patients for whom alternative treatments are inadequate
Approval date: April 24, 2017

Endari (new formulation)
(L-glutamine oral powder; Emmaus Medical)
Class/route: Amino acid; oral powder
New formulation: 5-g L-glutamine powder per paper-foil-plastic laminate packet
Indication: To reduce acute complications of sickle-cell disease in adults and pediatric patients aged ≥5 years; first treatment for sickle-cell disease in children aged ≥5 years
Approval date: July 7, 2017

Esbriet (new dosage form)
(Pirfenidone; Genentech)
Class/route: Pyridone; oral tablets
New dosage form: 267-mg, 534-mg, and 801-mg film-coated tablets
Existing dosage form: 267-mg capsules
Indication: For treatment of patients with idiopathic pulmonary fibrosis
Approval date: January 11, 2017

Gocovri (new dosage)
(Amantadine extended release; Adamas Pharmaceuticals)
Class/route: Dopamine agonist; oral capsules
New dosages: 68.5-mg and 137-mg extended-release capsules once daily
Existing dosage: 274-mg extended-release capsules once daily
Indication: For treatment of dyskinesia in patients with Parkinson’s disease who are receiving levodopa-based therapy, with or without concomitant dopaminergic drugs
Approval date: August 24, 2017

Harvoni (new patient population)
(Ledipasvir and sofosbuvir; Gilead Sciences)
Class/route: HCV NS5A inhibitor and HCV nucleotide analog NS5B polymerase inhibitor; oral tablets
New patient population: Children aged 12 to 17 years, or weighing at least 35 kg, with HCV infection genotype 1, 4, 5, or 6, without cirrhosis or with compensated cirrhosis
Existing patient populations: Adults with chronic HCV genotype 1, 4, 5, or 6 infection, without cirrhosis or with compensated cirrhosis; adults with genotype 1 infection and decompensated cirrhosis; adults with genotype 1 or 4 infection without cirrhosis or with compensated cirrhosis who had liver transplant
indications: For treatment of chronic HCV genotype 1, 4, 5, or 6 infection, without cirrhosis or with compensated cirrhosis; treatment of genotype 1 infection with decompensated cirrhosis, in combination with ribavirin; treatment, in combination with ribavirin, of genotype 1 or 4 infection without cirrhosis or with compensated cirrhosis in patients who had liver transplant
Approval date: April 7, 2017

Kalydeco (new patient population)
(Ivacaftor; Vertex Pharmaceuticals)
Class/route: CFTR potentiator; oral tablets
New patient population: Patients aged ≥2 years with cystic fibrosis and CFTR gene mutations
Existing patient population: Patients aged ≥6 years with cystic fibrosis and CFTR gene mutations
Indication: For treatment of cystic fibrosis in patients with CFTR gene mutations whose disease is responsive to ivacaftor therapy, based on clinical and/or in-vitro assay data
Approval date: August 1, 2017
See also III. New Indications listing

Latuda (new patient population)
(Lurasidone hydrochloride; Sunovion)
Class/route: Atypical antipsychotic; oral tablets
New patient population: Adolescents (aged 13-17 years) with schizophrenia
Existing patient population: Adults with schizophrenia or bipolar depression
indications: For treatment of schizophrenia in adults and adolescents; monotherapy for bipolar depression in adults and adolescents (aged 10-17 years); adjunctive therapy, with lithium or valproate, in adults with bipolar depression
Approval date: January 27, 2017

Linzess (new dosage form)
(Linaclotide; Allergan)
Class/route: Guanylate cyclase-C agonist; oral capsules
New dosage form: 72 mcg once daily for chronic idiopathic constipation
Existing dosage forms: 290 mcg once daily for adults with irritable bowel syndrome and constipation; 145 mcg once daily for adults with chronic idiopathic constipation
indications: For treatment of adults with irritable bowel syndrome and constipation; treatment of adults with chronic idiopathic constipation
Approval date: January 25, 2017

Lynparza (new dosage form)
(Olaparib; AstraZeneca)
Class/route: PARP inhibitor; oral tablets, oral capsules
New dosage form: Oral tablets
Existing dosage form: Oral capsules, which are being phased out and will only be available through the Lynparza Specialty Pharmacy Network
indications: For maintenance treatment of adults with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who have had a complete or partial response to platinum-based chemotherapy; treatment of HER2-negative metastatic breast cancer with deleterious or suspected deleterious germline BRCA mutation in patients who have received chemotherapy
Approval date: August 17, 2017
See also III. New Indications listing

Mydayis (new dosage form)
(Mixed salts of a single-entity amphetamine; Shire)
Class/route: Central nervous system stimulant; extended-release oral capsules
New dosage form: Once-daily extended-release, 12.5-mg, 25-mg, 37.5-mg, and 50-mg oral capsules that last up to 16 hours
Indication: For treatment of attention-deficit/ hyperactivity disorder in patients aged ≥13 years
Approval date: June 20, 2017

Mylotarg (new dosage/new patient population)
(Gemtuzumab ozogamicin; Pfizer)
Class/route: CD33-directed antibody-drug conjugate; intravenous injection
New dosage: 4.5-mg injection in 1 vial; dosing regimen varies by the patient’s age
New patient population: Pediatric patients aged ≥2 years with relapsed or refractory CD33-positive acute myeloid leukemia
indications: For treatment of adults with newly diagnosed CD33-positive acute myeloid leukemia, in combination with daunorubicin and cytarabine; treatment of relapsed or refractory CD33-positive acute myeloid leukemia in adults and in pediatric patients aged ≥2 years
Approval date: September 1, 2017
[NOTE: Mylotarg initially received accelerated approval in 2000 at a higher dose, for adults; the manufacturer removed it voluntarily from the market in 2010 because of serious adverse reactions at the higher dose]
See also III. New Indications listing

Narcan (new dosage form)
(Naloxone hydrochloride; Adapt Pharma)
Class/route: Opioid antagonist; intranasal spray
New dosage form: 2-mg dose
Existing dosage form: 4-mg dose
Indication: Emergency treatment of known or suspected opioid overdose
Approval date: January 24, 2017

Noctiva (new formulation)
(Desmopressin acetate; Serenity)
Class/route: Vasopressin analog; intranasal spray
New formulation: Proprietary low-dose formulation of desmopressin acetate administered through a patent­protected preservative-free intranasal delivery system
Indication: First FDA-approved treatment for adults who awaken at least 2 times per night to urinate as a result of nocturnal polyuria
Approval date: March 3, 2017

Norvir (new dosage form)
(Ritonavir; AbbVie)
Class/route: HIV protease inhibitor; oral tablets, oral solution, oral powder
New dosage form: Oral powder
Existing formulations: Oral tablets, oral solution
Indication: In combination with other antiretroviral agents, for treatment of pediatric patients with HIV-1 infection
Approval date: June 6, 2017

Praluent (new dosage)
(Alirocumab; Sanofi US/Regeneron Pharmaceuticals)
Class/route: PCSK9 inhibitor antibody; subcutaneous injection
New dosage: Once-monthly (every 4 weeks) 300-mg dose injection
Existing dosage: 75 mg once every 2 weeks administered subcutaneously
Indication: As an adjunct to diet and maximally tolerated statin therapy for treatment of adults with heterozygous familial hypercholesterolemia or clinical atherosclerotic cardiovascular disease in patients who require additional lowering of low-density lipoprotein cholesterol
Approval date: April 25, 2017

Rhofade (new formulation)
(Oxymetazoline hydrochloride; Allergan)
Class/route: Alpha1A adrenoceptor agonist; topical cream
New formulation: First-in-class topical treatment of persistent facial erythema associated with rosacea in adults
Indication: For topical treatment of persistent facial erythema associated with rosacea in adults
Approval date: January 19, 2017

RoxyBond (new formulation)
(Oxycodone hydrochloride; Inspirion Delivery Sciences)
Class/route: Opioid agonist; oral tablets
New formulation: First immediate-release opioid analgesic 5-mg, 15-mg, and 30-mg tablets with the SentryBond abuse-deterrent formulation
Existing formulation: Immediate-release 5-mg, 15-mg, and 30-mg tablets without abuse-deterrent properties
Indication: For management of pain severe enough to require an opioid analgesic in patients for whom alternative treatments are inadequate
Approval date: April 26, 2017

Sovaldi (new patient population)
(Sofosbuvir; Gilead Sciences)
Class/route: HCV nucleotide analog NS5B polymerase inhibitor; oral tablets
New patient population: Children aged 12 to 17 years or weighing ≥35 kg
Existing patient population: Adults aged ≥18 years
indications: For treatment of adults with genotype 1, 2, 3, or 4 chronic HCV infection without cirrhosis or with compensated cirrhosis as a component of a combination antiviral treatment regimen; treatment, in combination with ribavirin, of pediatric patients aged ≥12 years or weighing ≥35 kg with genotype 2 or 3 chronic HCV infection without cirrhosis or with compensated cirrhosis
Approval date: April 7, 2017

Stelara (new patient population)
(Ustekinumab; Janssen)
Class/route: Human interleukin-12 and -23 antagonist; subcutaneous or intravenous injection
New patient population: Adolescents aged ≥12 years with moderate-to-severe plaque psoriasis who are candidates for phototherapy or systemic therapy
indications: For treatment of adults with moderate-to-severe plaque psoriasis who are candidates for phototherapy or systemic therapy; for treatment of active psoriatic arthritis, alone or in combination with methotrexate; for treatment of moderately to severely active Crohn’s disease who did not respond to or were intolerant of treatment with immunomodulators or corticosteroids, but responded to a TNF blocker or did not respond to or were intolerant of treatment with ≥1 TNF blockers
Approval date: October 13, 2017

Sublocade (new dosage form)
(Buprenorphine extended-release; Indivior)
Class/route: Partial opioid agonist; oral tablets, oral sublingual film, subdermal implant, subcutaneous injection
New dosage form: First once-monthly injectable buprenorphine drug for the treatment of moderate-to-­severe opioid use disorder in adults who have initiated treatment with a transmucosal buprenorphine-containing drug
Existing dosage forms: Oral tablets, oral sublingual film, subdermal implant
Indication: For treatment of moderate-to-severe opioid use disorder in patients who have initiated treatment with a transmucosal buprenorphine-containing drug
Approval date: November 30, 2017

Symbicort (new patient population)
(Budesonide and formoterol fumarate dihydrate; AstraZeneca)
Class/route: Corticosteroid; long-acting bronchodilator
New patient population: Patients aged 6 to 12 years with asthma
Existing patient population: Patients aged ≥12 years with asthma
indications: Treatment of asthma in patients aged ≥6 years; treatment of airflow obstruction in adults with chronic obstructive pulmonary disease
Approval date: January 27, 2017

Symjepi (new delivery system)
(Epinephrine; Adamis Pharmaceuticals)
Class/route: Nonselective alpha and beta-adrenergic receptor agonist; intramuscular injection, subcutaneous injection
New delivery system: 0.3-mg prefilled single-dose syringe
Existing delivery system: USP autoinjector for intramuscular or subcutaneous use
Indication: For emergency treatment of allergic reactions (type I), including anaphylaxis
Approval date: June 15, 2017

Tepadina (new formulation)
(Thiotepa; Amneal Biosciences)
Class/route: Alkylating drug; intravenous, intracavitary, or intravesical injections
New formulation: 100-mg vial
Existing formulation: 15-mg vial
indications: To reduce the risk for graft rejection when used in conjunction with high-dose busulfan and cyclophosphamide as a preparative regimen for allogeneic hematopoietic progenitor (stem)-cell transplantation for pediatric patients with class 3 beta-thalassemia; for treatment of adenocarcinoma of the breast or ovary; for controlling intracavitary effusions secondary to diffuse or localized neoplastic diseases of various serosal cavities; for treatment of superficial papillary carcinoma of the urinary bladder
Approval date: April 27, 2017

Vantrela ER (new formulation)
(Hydrocodone bitartrate; Teva)
Class/route: Opioid agonist; oral tablets
New formulation: 15-mg, 30-mg, 45-mg, 60-mg, and 90-mg extended-release tablets with proprietary abuse-deterrent technology
Existing formulation: 20-mg, 30-mg, 40-mg, 60-mg, 80-mg, 100-mg, and 120-mg film-coated tablets without abuse-deterrent properties
Indication: For management of pain severe enough to require daily, around-the-clock, long-term opioid treatment in patients for whom alternative treatments are inadequate
Approval date: January 18, 2017

Zerviate (new formulation)
(Cetirizine ophthalmic solution; Nicox)
Class/route: Histamine-1 receptor antagonist; topical ophthalmic solution
New formulation: First topical cetirizine for application in the eye
Existing formulations: Oral solution, oral tablets, oral chewable tablets, oral capsules
Indication: For treatment of ocular itching associated with allergic conjunctivitis
Approval date: May 31, 2017

Zilretta (new formulation)
(Triamcinolone acetonide; Flexion Therapeutics)
Class/route: Synthetic corticosteroid; intra-articular injectable suspension
New formulation: First extended-release intra-articular injection for osteoarthritis knee pain
Existing formulations: Intranasal spray, intralesional injection, intramuscular injection, topical cream, topical ointment
Indication: For management of osteoarthritis pain of the knee
Approval date: October 6, 2017

CFTR indicates cystic fibrosis transmembrane conductance regulator; HCV, hepatitis C virus; PARP, poly (ADP-ribose) polymerase; PCSK9, proprotein convertase subtilisin kexin type 9; TNF, tumor necrosis factor.

 

V. New Biosimilars

Cyltezo (new biosimilar)
(Adalimumab-adbm; Boehringer Ingelheim)
Class/route: TNF blocker; subcutaneous injection
Reference drug: Humira (adalimumab)
indications: For treatment of adults with moderately to severely active rheumatoid arthritis, alone or in com­bination with methotrexate or other nonbiologic DMARDs; treatment of moderately to severely active polyarticular juvenile idiopathic arthritis in patients aged ≥4 years, alone or in combination with methotrexate; treatment of active psoriatic arthritis, alone or in combination with nonbiologic DMARDs; treatment of active ankylosing spondylitis; treatment of moderately to severely active Crohn’s disease in adults who have had an inadequate response to conventional therapy or who have lost response to or are intolerant of infliximab; treatment of moderately to severely active ulcerative colitis in adults who have had an inadequate response to immunosuppressant agents; treatment of moderate-to-­severe chronic plaque psoriasis in adults who are candidates for systemic therapy or phototherapy, and when other systemic therapies are less appropriate
Approval date: August 25, 2017

Ixifi (new biosimilar)
(Infliximab-qbtx; Pfizer)
Class/route: TNF blocker; intravenous injection
Reference drug: Remicade (infliximab)
indications: For treatment of patients aged ≥6 years with moderately to severely active Crohn’s disease who have had an inadequate response to conventional therapy; treatment of adults with moderately to severely active ulcerative colitis who have had an inadequate response to conventional therapy; treatment of patients with moderately to severely active rheumatoid arthritis, in combination with methotrexate; treatment of patients with active ankylosing spondylitis; treatment of patients with psori­atic arthritis; treatment of adults with chronic severe plaque psoriasis who are candidates for systemic therapy and when other systemic therapies are less appropriate
Approval date: December 13, 2017

Mvasi (new biosimilar)
(Bevacizumab-awwb; Amgen)
Class/route: VEGF-specific angiogenesis inhibitor; intravenous infusion
Reference drug: Avastin (bevacizumab)
indications: For treatment of patients with metastatic colorectal cancer, in combination with intravenous 5-fluorouracil–based chemotherapy, or, in patients whose disease progressed with a bevacizumab-containing regimen, in combination with fluoropyrimidine-­irinotecan– or fluoropyrimidine-oxaliplatin–based chemotherapy; unresectable, locally advanced, recurrent, or metastatic nonsquamous non–small-cell lung cancer, in combination with carboplatin and paclitaxel; treatment of glioblastoma in adults whose disease progressed after a single-agent therapy; treatment of metastatic renal-cell carcinoma, in combination with interferon alfa; treatment of persistent, recurrent, or metastatic cervical cancer, in combination with
paclitaxel and cisplatin or paclitaxel and topotecan
Approval date: September 14, 2017

Ogivri (new biosimilar)
(Trastuzumab-dkst; Mylan)
Class/route: HER2/neu receptor antagonist
Reference drug: Herceptin (trastuzumab)
indications: For adjuvant treatment of patients with HER2-overexpressing node-positive or node-negative breast cancer; treatment of patients with HER2-over­expressing metastatic breast cancer, in combination with paclitaxel or as a single agent; treatment of patients with HER2-overexpressing metastatic gastric or gastroesophageal junction adenocarcinoma, in combination with cisplatin and capecitabine or 5-fluorouracil
Approval date: December 1, 2017

Renflexis (new biosimilar)
(Infliximab-abda; Merck)
Class/route: TNF blocker; intravenous injection
Reference drug: Remicade (infliximab)
indications: For treatment of patients aged ≥6 years with moderately to severely active Crohn’s disease who have had an inadequate response to conventional therapy; treatment of adults with moderately to severely active ulcerative colitis who have had an inadequate response to conventional therapy; treatment of moderately to severely active rheumatoid arthritis, in combination with methotrexate; treatment of active ankylosing spondylitis; treatment of psoriatic arthritis; treatment of adults with chronic severe plaque psoriasis who are candidates for systemic therapy and when other systemic therapies are less appropriate
Approval date: April 21, 2017

DMARD indicates disease-modifying antirheumatic drug; TNF, tumor necrosis factor; VEGF, vascular endothelial growth factor.

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