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Pharmacy News

New Drug and Biological Product Approvals, 2012

[February 1, 2013, AJHP News]

Kate Traynor

BETHESDA, MD 14 Jan 2013—A total of 50 new molecular entities, vaccines, and blood-derived products won FDA marketing approval last year, compared with 37 in 2011. Highlights of several of the approved medications appear below. A full list of new products and their indications appears below.

First-in-class drugs. FDA’s Center for Drug Evaluation and Research (CDER) identified more than a dozen new drugs as the first in their class to be approved by the agency. First-in-class drugs are defined by CDER as those that "for example, use a new and unique mechanism of action for treating a medical condition."

First-in-class drugs approved during 2012 cover a wide range of illnesses.

Eisai Co.’s perampanel, or Fycompa, a noncompetitive α-amino-3-hydroxy-5-methyl-4-isoxazolepropionic acid glutamate receptor antagonist, was approved in October for the treatment of some forms of epilepsy.

The cystic fibrosis transmembrane conductance regulator potentiator ivacaftor, or Kalydeco, by Vertex Pharmaceuticals Inc., was approved in January. Ivacaftor is the first FDA-approved drug that targets an underlying cause of cystic fibrosis rather than treating its symptoms. The drug is indicated for use in patients whose cystic fibrosis transmembrane conductance regulator gene expresses the G551D mutation.

Teriflunomide, marketed as Aubagio by Genzyme, was approved in September for the treatment of relapsing forms of multiple sclerosis. The drug inhibits the production of the mitochondrial enzyme dihydroorotate dehydrogenase, whose mechanism of action against multiple sclerosis is unknown but may be related to a reduction in the number of lymphocytes circulating in the central nervous system.

Ocriplasmin was approved in October for the treatment of symptomatic vitreomacular adhesion. The drug, a recombinant truncated form of human plasmin, dissolves proteins in the eye that are responsible for the adhesion. Ocriplasmin is marketed as Jetrea by ThromboGenics Inc.

First-in-class cancer treatments include the orally administered drugs cabozantinib and vismodegib and omacetaxine mepesuccinate, an injectable drug.

Cabozantinib, sold as Cometriq by Exelixis Inc., inhibits multiple tyrosine kinases and is indicated for the treatment of advanced medullary thyroid cancer, a rare disease.

Vismodegib, sold as Erivedge by Genentech USA Inc., is the first drug approved specifically for the treatment of metastatic basal cell carcinoma and also the first FDA-approved drug to target the hedgehog signaling pathway.

The cephalotaxine ester omacetaxine mepesuccinate, a Teva drug marketed as Synribo, was approved as a second-line agent for the treatment of chronic myelogenous leukemia.

Also approved during 2012 was the recombinant carboxypeptidase glucarpidase, which is used to reverse methotrexate toxicity in patients with impaired kidney function. The drug is marketed as Voraxaze by BTG International Inc.

Choline C 11 injection, a positron emission tomography (PET) imaging agent for the detection of recurrent prostate cancer, was approved in September. The drug is produced by the Mayo Clinic, which is the only facility licensed by FDA to make the agent.

FDA in 2009 released requirements that all PET-agent production facilities must follow in order to sell their products in the United States. FDA gave manufacturers until June 2012 to submit drug-approval applications to the agency.

A second first-in-class imaging agent, Eli Lilly’s florbetapir F 18 injection, or Amyvid, was approved in April. The imaging agent is used to detect amyloid plaques in adults with Alzheimer’s disease or other causes of cognitive decline.

Biologics. Much of the approval action for biological products last year centered on influenza vaccines, with three new products licensed during the year.

MedImmune LLC’s FluMist Quadrivalent in February became the first U.S.-licensed seasonal influenza virus vaccine to cover four virus strains.

Traditional influenza virus vaccines contain antigens from two influenza virus type A strains and one type B strain, which is either of the so-called Victoria or Yamagata lineage. FluMist Quadrivalent, an intranasal vaccine, contains one type B strain from each lineage to increase the likelihood of protection against the type B viruses that circulate during the flu season.

Fluarix Quadrivalent, made by GlaxoSmithKline plc, in December became the second U.S.-licensed quadrivalent influenza virus vaccine. Fluarix Quadrivalent is administered by intramuscular injection.

Novartis AG in November received approval to market Flucelvax, the first U.S.-licensed trivalent seasonal influenza virus vaccine produced in a cell culture system. The federal government has supported the development of cell-based influenza vaccines as part of the nation’s pandemic response and planning activities.

2012 Pharmaceutical Approvals

New molecular entities and new biologicals, 2012a

Aclidinium bromide oral inhalation powder (Tudorza Pressair, Forest), for long-term maintenance therapy in patients with chronic obstructive pulmonary disease

Allogeneic cultured keratinocytes and fibroblasts in bovine collagen, cellular sheet for topical oral application, (Gintuit, Organogenesis), for the treatment of mucogingival conditions in adults

Apixaban oral tablets (Eliquis, Bristol-Myers Squibb), to reduce the risk of stroke and systemic embolism in patients with atrial fibrillation who do not have a prosthetic heart valve

Avanafil oral tablets (Stendra, Vivus), for the treatment of erectile dysfunction

Axitinib oral tablets (Inlyta, Pfizer), as second-line therapy in patients with advanced renal cell carcinoma that has not responded to previously administered systemic agents

Bedaquiline oral tablets (Sirturo, Janssen), as part of combination therapy for the treatment of multidrug-resistant pulmonary tuberculosis when other alternatives are unavailable

Bosutinib oral tablets (Bosulif, Pfizer), as a second-line agent for the treatment of chronic myelogenous leukemia in adults

Cabozantinib oral capsules (Cometriq, Exelixis), for the treatment of progressive, metastatic medullary thyroid cancer

Carfilzomib for i.v. injection (Kyprolis, Onyx), for the treatment of multiple myeloma in patients previously treated with at least two other therapies, including bortezomib and an immunomodulatory agent, whose disease progressed within 60 days after the last treatment

Choline C 11 injection (Mayo Clinic) for use during positron emission tomography to help detect recurrent prostate cancer

Crofelemer delayed-release oral tablets (Fulyzaq, Salix), for the symptomatic relief of noninfectious diarrhea in HIV-infected adults undergoing antiretroviral therapy

Elvitegravir, cobicistat, emtricitabine, and tenofovir disoproxil fumarate oral tablets (Stribild, Gilead), for the treatment of HIV-1 infection in adults who are new to antiretroviral treatment

Enzalutamide oral capsules (Xtandi, Astellas), for the treatment of advanced prostate cancer

Fibrin sealant patch, absorbable (Evarrest, Ethicon), to stop soft tissue bleeding during open retroperitoneal, intraabdominal, pelvic, and noncardiac thoracic surgery when suture, ligature, or cautery is ineffective or impractical

Florbetapir F 18 injection (Amyvid, Eli Lilly), for imaging amyloid plaques in adults whose cognitive impairment may be due to Alzheimer’s disease or another cause of cognitive decline

Glucarpidase for i.v. injection (Voraxaze, BTG International), for the treatment of toxic plasma methotrexate concentrations in patients whose kidneys are not eliminating the drug at a normal rate

Hematopoietic progenitor cells, cord blood for injection (Ducord, Duke University School of Medicine), for hematopoietic progenitor cell transplantation procedures in patients with inherited, acquired, or myeloablative-treatment-related diseases that affect the hematopoietic system

Hematopoietic progenitor cells, cord blood for injection (Hematopoietic progenitor cells, Clinimmune), for hematopoietic progenitor cell transplantation procedures in patients with inherited, acquired, or myeloablative-treatment-related diseases that affect the hematopoietic system

Icosapent ethyl oral capsules (Vascepa, Amarin), for the reduction of triglyceride levels in adults with hypertriglyceridemia

Immune globulin, human, for i.v. infusion (Bivigam, Biotest), for the treatment of primary humoral immunodeficiency

Influenza virus vaccine, inactivated, for i.m. injection (Fluarix Quadrivalent, Glaxo­SmithKline), for active immunization of children age three years or older and adults against influenza caused by influenza virus subtypes A and type B present in the vaccine

Influenza virus vaccine, inactivated, for i.m. injection (Flucelvax, Novartis), for active immunization of adults against influenza caused by influenza virus subtypes A and type B present in the vaccine

Influenza virus vaccine, live, intranasal spray (FluMist Quadrivalent, MedImmune), for active immunization of people 2–49 years of age against influenza caused by influenza virus subtypes A and type B present in the vaccine

Ingenol mebutate topical gel (Picato, Leo Pharma), for the topical treatment of actinic keratosis

Ivacaftor oral tablets (Kalydeco, Vertex), for the treatment of cystic fibrosis in patients 6 years of age or older whose cystic fibrosis transmembrane conductance regulator gene expresses the G551D mutation

Linaclotide oral capsules (Linzess, Forest), for the treatment of constipation-predominant irritable bowel syndrome and chronic idiopathic constipation in adults

Lomitapide oral capsules (Juxtapid, Aegerion), as part of a regimen for the treatment of patients with homozygous familial hypercholesterolemia

Lorcaserin hydrochloride oral tablets (Belviq, Eisai), for chronic weight management in obese and certain overweight adults, in conjunction with diet and exercise

Lucinactant intratracheal suspension (Surfaxin, Discovery), for the prevention of respiratory distress syndrome in premature infants

Meningococcal groups C and Y and Haemophilus b tetanus toxoid conjugate vaccine (Menhibrix, GlaxoSmithKline), for immunization against bacterial meningitis in infants and young children

Mirabegron extended-release oral tablets (Myrbetriq, Astellas), for the treatment of overactive bladder in adults

Ocriplasmin intravitreal injection (Jetrea, ThromboGenics), for the treatment of symptomatic vitreomacular adhesion

Omacetaxine mepesuccinate for s.c. injection (Synribo, Teva ), for the second-line treatment of chronic myelogenous leukemia in adults

Pasireotide diaspartate injection (Signifor, Novartis), for the treatment of Cushing’s disease in patients who are not candidates for or did not respond to pituitary surgery

Peginesatide injection (Omontys, Takeda and Affymax), for the treatment of anemia due to chronic kidney disease in adults undergoing dialysis

Perampanel oral tablets (Fycompa, Eisai), as adjunctive therapy for the treatment of partial-onset seizures in patients with epilepsy ages 12 years or older

Pertuzumab injection (Perjeta, Genentech), for the treatment of metastatic breast cancer, in combination with trastuzumab and docetaxel

Ponatinib oral tablets (Iclusig, Ariad), as a second-line treatment for all phases of chronic myelogenous leukemia and Philadelphia chromosome-positive acute lymphoblastic leukemia in adults

Raxibacumab for i.v. infusion (Abthrax, GlaxoSmithKline), for the treatment of adult and pediatric patients with inhalational anthrax due to Bacillus anthracis, in combination with appropriate antibacterial drugs, and for prophylaxis of inhalational anthrax when alternative therapies are not available or are not appropriate

Regorafenib oral tablets (Stivarga, Bayer and Onyx), for the treatment of metastatic colorectal cancer in patients previously treated with fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy, antivascular endothelial growth factor therapy, and, for patients with a wild-type Kras gene, antiepidermal growth factor receptor therapy

Sodium picosulfate and magnesium oxide and anhydrous citric acid for oral solution (Prepopik, Ferring), for bowel cleansing before colonoscopy procedures in adults

Tafluprost ophthalmic solution (Zioptan, Merck), for the reduction of elevated intraocular pressure in patients with open-angle glaucoma or ocular hypertension

Taliglucerase alfa for i.v. infusion (Elelyso, Pfizer), for the treatment of type 1 Gaucher disease in adults

Tbo-filgrastim for s.c. injection (Neutroval, Teva), for the stimulation of neutrophil production in adults who become neutropenic while undergoing chemotherapy for nonmyeloid cancers

Teduglutide for s.c. injection (Gattex, NPS), for the treatment of short bowel syndrome in adults dependent on parenteral nutrition

Teriflunomide oral tablets (Aubagio, Genzyme), for the treatment of relapsing forms of multiple sclerosis

Tofacitinib oral tablets (Xeljanz, Pfizer), for use as a second-line treatment for moderate-to-severe rheumatoid arthritis in adults

Varicella zoster immune globulin, human, for i.m. injection (Varizig, Cangene), for postexposure prophylaxis to reduce the severity of varicella in people at high risk for complications of the disease

Vismodegib oral capsules (Erivedge, Genentech), for the treatment of adults with locally advanced basal cell cancer who are not candidates for surgery or radiotherapy or whose cancer has metastasized

Ziv-aflibercept for i.v. infusion (Zaltrap, Regeneron and Sanofi), as part of a regimen for the treatment of metastatic colorectal cancer that is resistant to or has progressed after an oxaliplatin-containing regimen

a From information available on FDA's website, January 2013. This table does not include medications that have been previously marketed in the United States without an FDA-approved new drug application.

 

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