Special Feature Fifth Annual Payers' Guide to New FDA Approvals March 2014 Volume 7
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I. New Pharmaceuticals: New Molecular Entities (NMEs) and Biologic License Applications (BLAs) Approved
II. New Combinations, Formulations, Indications


I. New Pharmaceuticals: New Molecular Entities (NMEs) and Biologic License Applications (BLAs) Approved

Adempas (NME)
(Riociguat; Bayer HealthCare)
Class/route: Soluble guanylate cyclase stimulator; oral
Indications: For adults with chronic thromboembolic pulmonary hypertension (CTEPH) after surgery or patients with inoperable CTEPH to improve exercise capacity and World Health Organization (WHO) functional class; and patients with pulmonary arterial hypertension to improve their exercise capacity, improve their WHO functional class, and to delay clinical worsening
Approval considerations: Priority review, orphan, risk evaluation and mitigation strategy (REMS)

Anoro Ellipta (NME)
(Umeclidinium and vilanterol inhalation powder; GlaxoSmithKline)
Class/route: Anticholinergic and long-acting β2-adrenergic agonist combination; inhalation
Indication: For the long-term, once-daily maintenance treatment of airflow obstruction in patients with chronic obstructive pulmonary disease (COPD); not for the relief of acute bronchospasm or for the treatment of asthma

Aptiom (NME)
(Eslicarbazepine acetate; Sunovion Pharmaceuticals)
Class/route: Antiepileptic; oral
Indication: For partial-onset seizures as an add-on medication

Breo Ellipta (NME)
(Fluticasone furoate and vilanterol inhalation powder; GlaxoSmithKline)
Class/route: Corticosteroid and a long-acting β2-adrenergic agonist combination; inhalation
Indications: For the long-term, once-daily maintenance of airflow obstruction in patients with COPD, and to reduce exacerbations in patients with COPD; not for relief of acute bronchospasm or for the treatment of asthma

Brintellix (NME)
(Vortioxetine; Takeda Pharmaceuticals)
Class/route: Serotonergic antidepressant; immediate release; oral
Indication: For the treatment of major depressive disorder

Dotarem (NME)
(Gadoterate meglumine; Guerbet)
Class/route: Magnetic resonance imaging contrast media; intravenous
Indication: For use in magnetic resonance imaging of the brain, spine, and associated tissues of patients aged ≥2 years to detect and to visualize the area with disruption of the blood–brain barrier and/or normal vascularity
Approval consideration: Priority review

Duavee (NME)
(Conjugated estrogens/bazedoxifene; Pfizer)
Class/route: Sex hormone combination; oral
Indications: For women with a uterus and moderate-
to-severe hot flashes associated with menopause, and to prevent osteoporosis after menopause

Flublok (BLA)
(Influenza vaccine, recombinant hemagglutinin; Protein Sciences Corporation)
Class/route: Vaccine; intramuscular injection
Indication: For active immunization against disease caused by influenza virus subtypes A and type B contained in the vaccine in persons aged 18 through 49 years

Gazyva (NME)
(Obinutuzumab; Genentech)
Class/route: CD20-directed cytolytic monoclonal antibody; intravenous
Indication: For patients with previously untreated chronic lymphocytic leukemia (CLL) in combination with chlorambucil chemotherapy
Approval considerations: Priority review, breakthrough therapy, orphan drug

Gilotrif (NME)
(Afatinib; Boehringer Ingelheim)
Class/route: Kinase inhibitor; oral
Indication: For the first-line treatment of patients with metastatic non–small-cell lung cancer (NSCLC) whose tumors have epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 (L858R) substitution mutations as detected by an FDA-approved test
Approval considerations: Priority review, orphan drug, fast track

Imbruvica (NME)
(Ibrutinib; Pharmacyclics)
Class/route: Kinase inhibitor; oral
Indications: Treatment of patients with mantle-cell lymphoma who have received at least 1 previous therapy, and patients with CLL who had at least 1 previous therapy
Approval considerations: Priority review, breakthrough therapy, accelerated approval, fast track, orphan drug

Invokana (NME)
(Canagliflozin; Janssen)
Class/route: Sodium-glucose cotransporter-2 inhibitor; oral
Indication: For patients with type 2 diabetes in conjunction with diet and exercise to improve glycemic control

Kadcyla (NME)
(Ado-trastuzumab emtansine; Genentech)
Class/route: Antineoplastic agent; intravenous
Indication: Treatment of people with human EGFR 2–positive metastatic breast cancer who have received previous treatment with trastuzumab and a taxane, separately or in combination
Approval considerations: Priority review, fast track

Kcentra (BLA)
(Prothrombin complex concentrate, human; CSL Behring GmbH)
Class/route: Blood coagulation factor replacement; intravenous
Indication: For the urgent reversal of acquired coagulation factor deficiency induced by vitamin K antagonist (eg, warfarin) therapy in adult patients with acute major bleeding

Kynamro (NME)
(Mipomersen sodium; Genzyme/Sanofi)
Class/route: Antihyperlipidemic agent; subcutaneous injection
Indication: For patients with homozygous familial hypercholesterolemia as an adjunct to lipid-lowering medications and diet to reduce low-density lipoprotein cholesterol, apolipoprotein B, total cholesterol, and non–high-density lipoprotein cholesterol
Approval considerations: Orphan drug, REMS

Luzu (NME)
(Luliconazole; Medicis Pharmaceutical)
Class/route: Antifungal; topical
Indications: For the topical treatment of interdigital tinea pedis, tinea cruris, and tinea corporis caused by Trichophyton rubrum and Epidermophyton floccosum in patients aged ≥18 years

Lymphoseek (NME)
(Technetium Tc99m tilmanocept; Navidea Biopharmaceuticals)
Class/routes: Radioactive diagnostic agent; subcutaneous, intradermal, or peritumoral injection
Indication: For lymphatic mapping with a handheld gamma counter to assist in the localization of lymph nodes draining a primary tumor site in patients with breast cancer or melanoma

Mekinist (NME)
(Trametinib; GlaxoSmithKline)
Class/route: Kinase inhibitor; oral
Indication: As a single agent and in combination with dabrafenib, for patients with unresectable or metastatic melanoma with BRAF V600E or BRAF V600K mutations as detected by an FDA-approved test
Approval considerations: Priority review, fast track, orphan drug

Nesina (NME)
(Alogliptin; Takeda Pharmaceuticals)
Class/route: Dipeptidyl peptidase-4 inhibitor; oral
Indication: For adults with type 2 diabetes in conjunction with diet and exercise to improve glycemic control in adults with type 2 diabetes mellitus

NovoEight (BLA)
(Antihemophilic factor, recombinant; Novo Nordisk)
Class/route: rFactor VIII; intravenous
Indication: For adults and children with hemophilia A for the control and prevention of bleeding episodes, perioperative management, and routine prophylaxis to prevent/reduce bleeding episodes; not indicated for von Willebrand disease

Octaplas (BLA)
(Solvent/detergent treated, pooled human plasma; Octapharma USA)
Class/route: Blood modifier; intravenous infusion
Indications: Replacement of multiple coagulation factors in patients with acquired deficiencies resulting from liver disease and in patients undergoing cardiac surgery or liver transplant, and for plasma exchange in patients with thrombotic thrombocytopenic purpura

Olysio (NME)
(Simeprevir; Janssen Biotech)
Class/route: Protease inhibitor; oral
Indication: For the treatment of chronic hepatitis C infection as a component of a combination antiviral treatment regimen
Approval considerations: Priority review, fast track

Opsumit (NME)
(Macitentan; Actelion Pharmaceuticals)
Class/route: Endothelin receptor antagonist; oral
Indication: For the treatment of patients with pulmonary arterial hypertension to delay disease progression
Approval considerations: Orphan drug, REMS

Osphena (NME)
(Ospemifene; Shionogi)
Class/route: Estrogen agonist/antagonist; oral
Indication: To treat women experiencing moderate-to-severe dyspareunia due to menopause

Pomalyst (NME)
(Pomalidomide; Celgene)
Class/route: Antineoplastic and immunosuppressant; oral
Indication: Treatment of patients with multiple myeloma who received at least 2 previous therapies, including lenalidomide and bortezomib, and have demonstrated disease progression on or within 60 days of completion of the last therapy
Approval considerations: Accelerated approval, orphan drug, REMS

Ravicti (BLA)
(Glycerol phenylbutyrate; Hyperion Therapeutics)
Class/route: Urea cycle disorder agent; oral liquid
Indication: For use as a nitrogen-binding agent for the chronic management of patients aged ≥2 years with urea cycle disorders who cannot be managed by dietary protein restriction and/or amino acid supplementation alone; must be used with dietary protein restriction and, in some cases, dietary supplements
Approval considerations: Priority review, orphan drug

Rixubis (BLA)
(Coagulation factor IX, recombinant; Baxter Healthcare)
Class/route: Coagulation modifier; intravenous
Indication: For adults with hemophilia B to control/prevent bleeding episodes; perioperative management; and routine prophylaxis to prevent/reduce bleeding episodes

Sovaldi (NME)
(Sofosbuvir; Gilead Sciences)
Class/route: Nucleotide analog inhibitor; oral
Indication: For the treatment of chronic hepatitis C infection as a component of a combination antiviral treatment regimen
Approval considerations: Priority review, breakthrough therapy, fast track

Tafinlar (NME)
(Dabrafenib; GlaxoSmithKline)
Class/route: Multikinase inhibitor; oral
Indication: For patients with unresectable or metastatic melanoma with the BRAF V600E mutation as detected by an FDA-approved test; not indicated for the treatment of patients with wild-type BRAF melanoma
Approval considerations: Fast track, orphan drug

Tecfidera (NME)
(Dimethyl fumarate; Biogen Idec)
Class/route: Selective immunosuppressant; oral
Indication: For patients with relapsing forms of multiple sclerosis

Tivicay (NME)
(Dolutegravir; ViiV Healthcare)
Class/route: Integrase strand transfer inhibitor; oral
Indication: For the treatment of HIV-1 infection in adults and children aged ≥12 years, weighing ≥40 kg, in combination with other antiretroviral agents
Approval considerations: Priority review, fast track

TOBI Podhaler (BLA)
(Tobramycin inhalation powder; Novartis)
Class/route: Aminoglycoside antibiotic; inhalation
Indication: For the management of cystic fibrosis patients with Pseudomonas aeruginosa

Tretten (BLA)
(Coagulation factor XIII A-subunit, recombinant; Novo Nordisk)
Class/route: Anticoagulation factor; intravenous
Indication: For routine prophylaxis of bleeding in patients with congenital factor XIII A-subunit deficiency; not for use in patients with congenital factor XII B-subunit
Approval consideration: Orphan drug

Velphoro (BLA)
(Sucroferric oxyhydroxide; Vifor Pharma)
Class/route: Iron-based, calcium-free phosphate binder; oral (chewable) tablet
Indication: For control of hyperphosphatemia in patients with chronic kidney disease

Vizamyl (NME)
(Flutemetamol F 18; GE Healthcare)
Class/route: Radioactive diagnostic agent; intravenous
Indication: For positron emission tomography imaging of the brain to estimate β-amyloid neuritic plaque density in adults with cognitive impairment who are being evaluated for Alzheimer’s disease and other causes of cognitive decline

Xofigo (NME)
(Radium Ra 223 dichloride; Bayer HealthCare)
Class/route: Diagnostic radiopharmaceutical; intravenous
Indication: For patients with symptomatic metastatic castration-resistant prostate cancer, symptomatic bone metastases, and no known visceral metastatic disease
Approval considerations: Priority review, fast track


II. New Combinations, Formulations, Indications

Abilify Maintena (new formulation)
(Aripiprazole; Otsuka Pharmaceutical)
Class/route: Atypical antipsychotic; intramuscular injection
New formulation: Extended-release injectable suspension for intramuscular injection; already available in oral form
Indications: For the treatment of schizophrenia; acute treatment of manic or mixed episodes associated with bipolar I disorder as monotherapy and as an adjunct to lithium or valproate; maintenance treatment of bipolar I disorder, both as monotherapy and as an adjunct to lithium or valproate; adjunctive treatment of major depressive disorder; treatment of irritability associated with autistic disorder; acute treatment of agitation associated with schizophrenia or bipolar I disorder

Abraxane (new indication)
(Paclitaxel protein-bound particles for injectable suspension [albumin bound]; Celgene)
Class/route: Antineoplastic agent; intravenous infusion
New indication: First-line treatment of patients with metastatic adenocarcinoma of the pancreas, in combination with gemcitabine; already indicated for metastatic breast cancer after failure of combination chemotherapy for metastatic disease or relapse within 6 months of adjuvant chemotherapy, and for locally advanced or metastatic non–small-cell lung cancer (NSCLC) as first-line treatment in combination with carboplatin in patients who are not candidates for curative surgery or radiation therapy
Approval considerations: Priority review, orphan drug

Aciphex Sprinkle
(new indication)
(Rabeprazole sodium; Eisai)
Class/route: Proton pump inhibitor; oral
New indication: For the treatment of gastroesophageal reflux disease (GERD) in pediatric patients aged 1 to 11 years; already indicated for erosive or ulcerative GERD, maintenance of healing of erosive or ulcerative GERD, treatment of symptomatic GERD, healing of duodenal ulcers, Helicobacter pylori eradication to reduce the risk of duodenal ulcer recurrence, treatment of pathologic hypersecretory conditions (including Zollinger-Ellison syndrome), and in adolescents aged ≥12 years for short-term treatment of symptomatic GERD
Approval consideration: Priority review

Actemra (new formulation, new indication)
(Tocilizumab; Genentech)
Class/route: Humanized antihuman interleukin-6 receptor antagonist monoclonal antibody; subcutaneous
New formulation: Subcutaneous injection; already available as intravenous
New indication: For the treatment of patients aged ≥2 years with active polyarticular juvenile idiopathic arthritis
Indications: For the treatment of adults with moderately to severely active rheumatoid arthritis (RA) who have used ≥1 disease-modifying antirheumatic drugs, and patients aged ≥2 years with active systemic juvenile idiopathic arthritis

Amitiza (new indication)
(Lubiprostone; Sucampo Pharma Americas)
Class/route: Chloride channel activator; oral
New indication: For the treatment of opioid-induced constipation in adults with chronic noncancer pain; already indicated for the treatment of chronic idiopathic constipation in adults and of irritable bowel syndrome with constipation in women aged ≥18 years
Approval consideration: Priority review

Astagraf XL (new formulation)
(Tacrolimus extended-release capsules;
Astellas Pharma US)
Class/route: Calcineurin inhibitor immunosuppressant; oral
New formulation: Extended-release capsules
Indication: For the prophylaxis of organ rejection in patients receiving a kidney transplant with mycophenolate mofetil and corticosteroids, with or without basiliximab induction

Botox (new indication)
(OnabotulinumtoxinA; Allergan)
Class/routes: Acetylcholine release inhibitor and neuromuscular blocking agent; intramuscular, intradetrusor, and intradermal
New indications: Treatment of overactive bladder with symptoms of urge urinary incontinence, urgency, and frequency, in adults who have an inadequate response to or are intolerant of an anticholinergic medication; and treatment of urinary incontinence resulting from detrusor overactivity associated with a neurologic condition in adults who have an inadequate response to or are intolerant of an anticholinergic medication; already indicated for the prophylaxis of headaches in adults with chronic migraine, upper limb spasticity in adults, cervical dystonia in adults to reduce the severity of abnormal head position and neck pain, severe axillary hyperhidrosis that is inadequately managed by topical agents in adults, blepharospasm associated with dystonia in patients aged ≥12 years, and strabismus in patients aged ≥12 years

Botox Cosmetic (new indication)
(OnabotulinumtoxinA; Allergan)
Class/route: Acetylcholine release inhibitor and neuromuscular blocking agent; intramuscular injection
New indication: Temporary improvement in the appearance of moderate-to-severe lateral canthal lines associated with orbicularis oculi activity in adults; already indicated for the temporary improvement in the appearance of moderate-to-severe glabellar lines associated with corrugator and/or procerus muscle activity in adults

Brisdelle (new formulation)
(Paroxetine; Noven Therapeutics)
Class/route: Selective serotonin reuptake inhibitor; oral
New formulation: Once-daily 7.5-mg capsules for oral use
Indication: For the treatment of moderate-to-severe vasomotor symptoms associated with menopause

Cimzia (new indication)
(Certolizumab pegol; UCB)
Class/route: Tumor necrosis factor blocker; subcutaneous
New indication: For the treatment of adults with active psoriatic arthritis and for adults with active ankylosing spondylitis; previously indicated for reducing the signs and symptoms of Crohn’s disease and maintaining clinical response in adults with moderately to severely active disease who have had an inadequate response to conventional therapy, and for the treatment of adults with moderately to severely active RA

Delzicol (new formulation)
(Mesalamine; Warner Chilcott)
Class/route: Aminosalicylate; oral
New formulation: Delayed-release capsule
Indication: For the treatment of mildly to moderately active ulcerative colitis and for the maintenance of remission of ulcerative colitis

Diclegis (new formulation)
(Doxylamine succinate and pyridoxine hydrochloride; Duchesnay USA)
Class/route: Combination of an antihistamine and a vitamin B6 analog; oral
New formulation: Fixed-dose combination in a delayed-release tablet
Indication: For the treatment of nausea and vomiting in pregnancy in women who do not respond to conservative management

Epiduo (new indication)
(Adapalene and benzoyl peroxide; Galderma Laboratories)
Class/route: Retinoid/oxidizing agent; topical
New indication: For the treatment of acne vulgaris in patients aged ≥9 years

Exelon Patch (new indication)
(Rivastigmine; Novartis)
Class/route: Acetylcholinesterase inhibitor; transdermal patch
New indication: For the treatment of patients with severe Alzheimer’s disease; already indicated for the treatment of mild-to-moderate dementia of the Alzheimer’s type and mild-to-moderate dementia associated with Parkinson’s disease

Exjade (new indication)
(Deferasirox; Novartis)
Class/route: Iron chelator; oral
New indication: For the treatment of chronic iron overload in patients aged ≥10 years with non–transfusion-dependent thalassemia syndromes and with a liver iron concentration of ≥5 mg of iron per gram of dry weight and a serum ferritin >300 mcg/L; previously indicated for the treatment of chronic iron overload resulting from blood transfusions in patients aged ≥2 years

Fetzima (new enantiomer)
(Levomilnacipran, a new enantiomer of milnacipran; Forest Laboratories)
Class/route: Serotonin and norepinephrine reuptake inhibitor; extended-release capsules, oral
Indication: Major depressive disorder in adults

Gleevec (new indication)
(Imatinib mesylate; Novartis)
Class/route: Kinase inhibitor; oral
New indication: Pediatric patients with newly diagnosed Philadelphia chromosome–positive acute lymphoblastic leukemia in combination with chemotherapy

Ilaris (new indication)
(Canakinumab; Novartis)
Class/route: Interleukin-1β blocker; subcutaneous injection
New indication: To treat active systemic juvenile idopathic arthritis in patients aged ≥2 years; already indicated for the treatment of cryopyrin-associated periodic syndromes, including familial cold autoinflammatory syndrome and Muckle-­Wells syndrome

Injectafer (new formulation)
(Ferric carboxymaltose injection; American Regent)
Class/route: Iron replacement; intravenous
New formulation: High-dose nondextran intravenous iron
Indication: For the treatment of iron-deficiency anemia in adults who have intolerance to oral iron or who have had an unsatisfactory response to oral iron, and adults who have nondialysis-dependent chronic kidney disease

Karbinal ER (new formulation)
(Carbinoxamine maleate; Tris Pharma)
Class/route: Histamine receptor blocker; oral
New formulation: Sustained-release oral suspension
Indications: For the symptomatic treatment of seasonal and perennial allergic rhinitis, vasomotor rhinitis, allergic conjunctivitis resulting from inhalant allergens and foods, mild uncomplicated allergic skin manifestations of urticaria and angioedema, and dermatographism; as therapy for anaphylactic reactions adjunctive to epinephrine and other standard measures after the acute manifestations have been controlled; and for the amelioration of the severity of allergic reactions to blood or plasma

Kazano (new combination)
(Alogliptin and metformin hydrochloride; Takeda Pharmaceuticals)
Class/route: Dipeptidyl peptidase-4 inhibitor and a biguanide; oral
Indication: For adults with type 2 diabetes mellitus as an adjunct to diet and exercise to improve glycemic control

Khedezla (new formulation)
(Desvenlafaxine; Osmotica Pharmaceutical)
Class/route: Serotonin and norepinephrine reuptake inhibitor; oral
New formulation: Extended-release tablets
Indication: For the treatment of major depressive disorder

Latuda (new indications)
(Lurasidone hydrochloride; Sunovion Pharmaceuticals)
Class/route: Atypical antipsychotic; oral
New indications: Depressive episodes associated with bipolar I disorder as monotherapy and as adjunctive therapy with lithium or valproate; already indicated for schizophrenia

Liptruzet (new combination)
(Ezetimibe and atorvastatin; Merck)
Class/route: Cholesterol absorption inhibitor and an HMG-CoA reductase inhibitor; oral
Indications: As adjunctive therapy to diet to reduce elevated total cholesterol, low-density lipoprotein cholesterol, apolipoprotein B, triglycerides, and non–high-density lipoprotein cholesterol (HDL-C), and to increase HDL-C in patients with primary (heterozygous familial and nonfamilial) hyperlipidemia or mixed hyperlipidemia; to reduce elevated total cholesterol and low-density lipoprotein cholesterol in patients with homozygous familial hypercholesterolemia as an adjunct to other lipid-lowering treatments

Mirvaso (new dosage form)
(Brimonidine; Galderma Laboratories)
Class/route: Alpha-adrenergic agonist; topical
New dosage form: Topical gel 0.33%
Indication: For the topical treatment of persistent (nontransient) facial erythema of rosacea in adults aged ≥18 years

Mycamine (new indication)
(Micafungin sodium; Astellas Pharma US)
Class/route: Echinocandin antifungal agent; intravenous infusion
New indication: For adults and pediatric patients aged ≥4 months to treat candidemia, acute disseminated candidiasis, Candida peritonitis and abscesses; patients with esophageal candidiasis; prophylaxis of Candida infections in patients undergoing hematopoietic stem-cell transplantation (HSCT)

Nexavar (new indication)
(Sorafenib; Bayer HealthCare)
Class/route: Kinase inhibitor; oral
New indication: For patients with locally recurrent or metastatic, progressive, differentiated thyroid carcinoma refractory to radioactive iodine treatment; already indicated for patients with unresectable hepatocellular carcinoma and patients with advanced renal-cell carcinoma and liver cancer
Approval consideration: Priority review, orphan drug

Noxafil (new formulation)
(Posaconazole; Merck)
Class/route: Azole antifungal agent; oral
New formulation: Delayed-release tablets; already available as an oral suspension
Indications: For the prophylaxis of invasive Aspergillus and Candida infections in patients aged ≥13 years who are at high risk of developing these infections as a result of being severely immunocompromised, such as HSCT recipients with graft-versus-host disease or those with hematologic malignancies with prolonged neutropenia from chemotherapy

Nymalize (new formulation)
(Nimodipine; Arbor Pharmaceuticals)
Class/route: Dihydropyridine calcium channel blocker; oral
New formulation: Oral solution; already available in capsules
Indication: For the improvement of neurologic outcome by reducing the incidence and severity of ischemic deficits in adults with subarachnoid hemorrhage from ruptured intracranial berry aneurysms regardless of their postictus neurologic condition (ie, Hunt and Hess Grades I-V)

Orenitram (new formulation)
(Treprostinil; United Therapeutics)
Class/route: Prostacyclin vasodilator; oral
New formulation: Extended-release oral tablets; already available as subcutaneous or intravenous solution and as an inhaled solution
Indication: For the treatment of pulmonary arterial hypertension (WHO Group 1) to improve exercise capacity

Oseni (new combination)
(Alogliptin and pioglitazone; Takeda Pharmaceuticals)
Class/route: Dipeptidyl peptidase-4 inhibitor and thiazolidinedione; oral
Indication: As an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes mellitus

Otrexup (new formulation)
(Methotrexate; Antares Pharma)
Class/new route: Folate analog metabolic inhibitor; subcutaneous
Indications: For the management of patients with severe, active RA and polyarticular juvenile idiopathic arthritis who are intolerant of or had an inadequate response to first-line therapy, and for the symptomatic control of severe, recalcitrant, disabling psoriasis in adults who are not adequately responsive to other forms of therapy

Perjeta (new indication)
(Pertuzumab; Genentech)
Class/route: HER2/neu receptor antagonist; intravenous injection
New indication: Use in combination with trastuzumab and docetaxel as neoadjuvant treatment of patients with HER2-positive, locally advanced, inflammatory, or early-stage breast cancer (either >2 cm in diameter or node positive) as part of a complete treatment regimen for early breast cancer; already indicated for use in combination with trastuzumab and docetaxel for treatment of patients with HER2-positive metastatic breast cancer who have not received previous anti-HER2 therapy or chemotherapy for metastatic disease
Approval consideration: Priority review

Prevnar 13 (new indication)
(Pneumococcal 13-valent conjugate vaccine [diphtheria CRM197 protein]; Pfizer)
Class/route: Vaccine; intramuscular injection
New indication: In children aged 6 years through 17 years (before the 18th birthday), for active immunization for the prevention of invasive disease caused by Streptococcus pneumoniae serotypes 1, 3, 4, 5, 6A, 6B, 7F, 9V, 14, 18C, 19A, 19F, and 23F; already indicated for children aged 6 weeks through 5 years (before the 6th birthday), for the active immunization for the prevention of invasive disease caused by S pneumoniae serotypes 1, 3, 4, 5, 6A, 6B, 7F, 9V, 14, 18C, 19A, 19F, and 23F; for active immunization for the prevention of otitis media caused by S pneumoniae serotypes 4, 6B, 9V, 14, 18C, 19F, and 23F; in adults aged ≥50 years, for active immunization for the prevention of pneumonia and invasive disease caused by S pneumoniae serotypes 1, 3, 4, 5, 6A, 6B, 7F, 9V, 14, 18C, 19A, 19F, and 23F

Procysbi (new formulation)
(Cysteamine bitartrate; Raptor Pharmaceuticals)
Class/route: Cystine-depleting agent; oral
New formulation: Delayed-release capsules
Indication: For the management of nephropathic cystinosis in adults and children aged ≥6 years
Approval consideration: Orphan drug

Prolensa (new formulation)
(Bromfenac ophthalmic solution; Bausch & Lomb)
Class/route: Nonsteroidal anti-inflammatory drug; topical ophthalmic solution
New formulation: Once-daily bromfenac 0.07% topical ophthalmic solution
Indications: For the treatment of postoperative inflammation and for the reduction of ocular pain in patients who have undergone cataract surgery

Quartette (new formulation)
(Levonorgestrel/ethinyl estradiol and ethinyl estradiol; Teva Pharmaceuticals)
Class/route: Estrogen/progestin combined oral contraceptive; oral
New formulation: 91-day oral tablet regimen
Indication: For use by women to prevent pregnancy

Revlimid (new indication)
(Lenalidomide; Celgene)
Class/route: Thalidomide analog; oral
New indication: For patients with mantle-cell lymphoma whose disease has relapsed or progressed after 2 prior therapies; already indicated for multiple myeloma, in combination with dexamethasone, in patients who have received at least1 prior therapy; transfusion-dependent anemia due to low- or intermediate-1–risk myelodysplastic syndromes associated with a deletion 5q abnormality with or without additional cytogenetic abnormalities

Simbrinza (new combination)
(Brinzolamide/brimonidine tartrate; Alcon Laboratories)
Class/route: Fixed-dose combination of a carbonic anhydrase inhibitor and an alpha-2 adrenergic receptor agonist; ophthalmic suspension; the only beta blocker–free treatment for intraocular pressure
Indication: For the reduction of elevated intraocular pressure in patients with primary open-angle glaucoma or ocular hypertension

Simponi (new indication)
(Golimumab; Janssen Biotech)
Class/route: Tumor necrosis factor blocker; subcutaneous injection
New indication: Moderate-to-severe ulcerative colitis with an inadequate response or intolerant to previous treatment or requiring continuous steroid therapy; already indicated for moderately to severely active RA in combination with methotrexate, active psoriatic arthritis alone or in combination with methotrexate, and active ankylosing spondylitis

Simponi Aria (new formulation)
(Golimumab; Janssen Biotech)
Class/route: Tumor necrosis factor blocker; intravenous
New formulation: Intravenous infusion; already available as subcutaneous injection
Indication: For the treatment of adults with moderately to severely active RA in combination with methotrexate

Sitavig (new formulation)
(Acyclovir; BioAlliance Pharma)
Class/route: Synthetic purine nucleoside analog; oral
New formulation: Buccal tablets
Indication: For the treatment of recurrent herpes labialis (cold sores) in immunocompetent adults

Skyla (new formulation)
(Levonorgestrel-releasing intrauterine system; Bayer HealthCare)
Class/route: Progestin-containing intrauterine system; intrauterine
New formulation: Intrauterine device that releases 13.5 mg of levonorgestrel slowly for up to 3 years
Indication: For the prevention of pregnancy for up to 3 years

Stelara (new indication)
(Ustekinumab; Janssen Biotech)
Class/route: Human interleukin-12 and interleukin-23 antagonist; subcutaneous
New indication: For adults with active psoriatic arthritis, alone or in combination with methotrexate; already indicated for adults with moderate-to-severe plaque psoriasis who are candidates for phototherapy or systemic therapy

Stivarga (new indication)
(Regorafenib; Bayer HealthCare)
Class/route: Kinase inhibitor; oral
New indication: Locally advanced unresectable or metastatic gastrointestinal stromal tumor previously treated with imatinib mesylate and sunitinib malate; already indicated for metastatic colorectal cancer previously treated with fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy, an antivascular endothelial growth factor therapy, and, if KRAS
wild-type, an anti–epidermal growth factor receptor (EGFR) therapy
Approval considerations: Priority review, orphan drug

Sustiva (new indication)
(Efavirenz; Bristol-Myers Squibb)
Class/route: Nonnucleoside reverse transcriptase inhibitor; oral
New indication: In combination with other antiretroviral agents for the treatment of human immunodeficiency virus type 1 infection in adults and in pediatric patients aged ≥3 months and weighing ≥3.5 kg using a “capsule sprinkle” method of administration (contents of the open capsule sprinkled into a small amount of food or formula)

Tarceva (new indication)
(Erlotinib; Astellas Pharma/Genentech)
Class/route: Kinase inhibitor; oral
New indication: First-line treatment of patients with metastatic NSCLC whose tumors have EGFR exon 19 deletions or exon 21 (L858R) substitution mutations as detected by a US Food and Drug Administration–approved test; already indicated for maintenance treatment of patients with locally advanced or metastatic NSCLC whose disease has not progressed after 4 cycles of platinum-based first-line chemotherapy; the treatment of locally advanced or metastatic NSCLC after failure of at least 1 previous chemotherapy regimen; the first-line treatment of patients with locally advanced, unresectable or metastatic pancreatic cancer in combination with gemcitabine; and in combination with gemcitabine for the first-line treatment of patients with locally advanced unresectable or metastatic pancreatic cancer

Topicort (new formulation)
(Desoximetasone; Taro Pharmaceutical Industries)
Class/route: Corticosteroid; topical
New formulation: Topical spray, 0.25%; already available as a cream, gel, and ointment
Indication: For the treatment of plaque psoriasis in patients aged ≥18 years

Trokendi XR (new formulation)
(Topiramate; Supernus Pharmaceuticals)
Class/route: Antiepileptic; oral
New formulation: Once-daily extended-release capsules
Indications: For initial monotherapy in patients aged ≥10 years with partial-onset or primary generalized tonic-clonic seizures, as adjunctive therapy in patients aged ≥6 years with partial-onset or primary generalized tonic-clonic seizures, and for adjunctive therapy in patients aged ≥6 years with seizures associated with Lennox-Gastaut syndrome

Uceris (new formulation)
(Budesonide; Santarus)
Class/route: Glucocorticosteroid; oral
New formulation: 9-mg extended-release tablets
Indication: For the induction of remission in patients with active, mild-to-moderate ulcerative colitis

Valchlor (new formulation)
(Mechlorethamine; Actilion)
Class/route: Alkylating drug; topical
New formulation: Topical gel, 0.016%
Indication: For the topical treatment of Stage IA and IB mycosis fungoides–type cutaneous T-cell lymphoma in patients who have received previous skin-directed therapy
Approval consideration: Orphan drug

Varithena (new formulation)
(Polidocanol; BTG)
Class/route: Sclerosing agent; intravenous
New formulation: Injectable foam for intravenous use under ultrasound guidance
Indications: For the treatment of incompetent great saphenous veins, accessory saphenous veins, and visible varicosities of the great saphenous vein system above and below the knee; improves the symptoms of superficial venous incompetence and the appearance of visible varicosities

Vibativ (new indication)
(Telavancin; Theravance)
Class/route: Lipoglycopeptide antibacterial; intravenous
New indication: Hospital-acquired and ventilator-associated bacterial pneumonia caused by susceptible isolates of Staphylococcus aureus when alternative treatment is not suitable; already indicated for complicated skin and skin structure infections

Vituz (new formulation)
(Hydrocodone bitartrate and chlorpheniramine maleate; Hawthorn Pharmaceuticals)
Class/route: A combination of an antitussive and a histamine-1 receptor antagonist; oral
New formulation: Oral solution
Indications: For the relief of cough and symptoms associated with upper respiratory allergies or a common cold in adults aged ≥18 years

Xgeva (new indication)
(Denosumab; Amgen)
Class/route: Ligand inhibitor; subcutaneous
New indication: For the treatment of adults and skeletally mature adolescents with giant-cell tumor of bone that is unresectable or where surgical resection is likely to result in severe morbidity; already indicated for the prevention of skeletal-related events in patients with bone metastases from solid tumors
Approval consideration: Priority review, orphan drug

Xiaflex (new indication)
(Collagenase clostridium histolyticum; Auxilium Pharmaceuticals)
Class/route: Bacterial collagenase; intralesional injection
New indication: For the treatment of adult men with Peyronie’s disease with a palpable plaque and curvature deformity of at least 30 degrees at the start of therapy; already indicated for adults with Dupuytren’s contracture with a palpable cord

Zecuity (new formulation)
(Sumatriptan iontophoretic transdermal system; NuPathe)
Class/route: Serotonin 1b/1d receptor agonist; transdermal
New formulation: Single-use, battery-powered patch
Indication: For the acute treatment of migraine with or without aura in adults

Zohydro ER (new formulation)
(Hydrocodone bitartrate; Zogenix)
Class/route: Opioid agonist; oral
New 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

Zubsolv (new formulation)
(Buprenorphine and naloxone; Orexo AB)
Class/route: Partial opioid agonist; sublingual
New formulation: Sublingual tablet
Indication: For the maintenance treatment of opioid dependence
Approval consideration: Risk evaluation and mitigation strategy

Last modified: April 17, 2014
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