FDA drug approvals-July 2014

July 1, 2014

FDA drug approvals, priority review, breakthrough designation, fast-track designations, orphan drug designations, first-time generic approval

Actions in brief

Tavaborole (Kerydin, Anacor Pharmaceuticals) topical solution, 5% was approved, which is the first oxaborole antifungal approved for the topical treatment of onychomycosis of the toenails.

Coagulation Factor VIIa [Recombinant]) (NovoSeven RT, Novo Nordisk) as the first recombinant treatment for bleeding episodes and perioperative management in patients with Glanzmann's Thrombasthenia (GT) with refractoriness to platelet transfusions, with or without antibodies to platelets

Belinostat (Beleodaq, Spectrum Pharmaceuticals) was approved to treat patients with peripheral T-cell lymphoma (PTCL), a rare and fast-growing type of non-Hodgkin lymphoma.

Insulin human Inhalation Powder (Afrezza, MannKind), a rapid-acting inhaled insulin, was approved to improve glycemic control in adults with diabetes mellitus.

A New Drug Application was approved for phenylephrine hydrochloride (Vazculep, Flamel Technologies) for the treatment of clinically important hypotension resulting primarily from vasodilation in the setting of anesthesia.

Tedizolid phosphate (Sivextro, Cubist Pharmaceuticals) was approved for the treatment of adults with acute bacterial skin and skin structure infections (ABSSSI) caused by certain susceptible bacteria, including Staphylococcus aureus (including methicillin-resistant strains (MRSA) and methicillin-susceptible strains), various Streptococcus species, and Enterococcus faecalis. Sivextro is available for intravenous and oral use.

Gadobutrol (Gadavist, Bayer HealthCare) injection was approved for intravenous use with MRI of the breast to assess the presence and extent of malignant breast disease.

Cyanocobalamin, USP (Nascobal, Strativa Pharmaceuticals) nasal spray single-use device was approved for the maintenance of normal hematologic status in pernicious anemia patients who are in remission following intramuscular vitamin B12 therapy and who have no nervous system involvement.

A new use for Lymphoseek (technetium 99m tilmanocept, Navidea Pharmaceuticals) Injection, a radioactive diagnostic imaging agent used to help doctors determine the extent of squamous cell carcinoma has spread in the body’s head and neck region, was approved. With this approval, Lymphoseek can now be used to guide testing of lymph nodes closest to a primary tumor for cancer, called a “sentinel” lymph node biopsy, in patients with cancer of the head and neck. This new indication will allow for the option of more limited lymph node surgery in patients with sentinel nodes negative for cancer.

Buprenorphine and naloxone (Bunavail, BioDelivery Sciences International) was approved for the maintenance treatment of opioid dependence and should be used as part of a complete treatment plan to include counseling and psychosocial support. 

Antihemophilic Factor (Recombinant), Fc Fusion Protein (Eloctate, Biogen Idec) was approved for the control and prevention of bleeding episodes, perioperative management and routine prophylaxis in adults and children with hemophilia A. This is the first recombinant hemophilia A therapy with prolonged circulation in the body. It is the only treatment for hemophilia A to reduce the frequency of bleeding episodes with prophylactic infusions every three to five days, offering people with hemophilia A the potential to extend the interval between prophylactic infusions.

The indication for rasagiline tablets (Azilect, Teva) was expanded from monotherapy and adjunct to levodopa to now include adjunct to dopamine agonists (DAs). The expanded indication reflects that Azilect can be used alone or in combination with other Parkinson’s disease medications.

 

Priority review

Ceftolozane/tazobactam (Cubist) for the treatment of complicated urinary tract Infections and complicated intra-abdominal infections. 

Investigational, all-oral, interferon-free regimen (Abbvie) for the treatment of adult patients with chronic genotype 1 hepatitis C virus.

Breakthrough designation

BiTE Antibody blinatumomab (Amgen) for the treatment of adults with Philadelphia-negative (Ph-) relapsed/refractory B-precursor acute lymphoblastic leukemia.

Idarucizumab (Boehringer Ingelheim), an investigational fully humanized antibody fragment, or Fab, as a specific antidote for Pradaxa (dabigatran etexilate mesylate).

Fast-track designations

HT-100 (Akashi Therapeutics), delayed-release halofuginone, to reduce fibrosis and inflammation and promote healthy muscle regeneration in boys with Duchenne muscular dystrophy.

DNX-2401 (DNAtrix), a conditionally replication-competent adenovirus for the treatment of several cancer indications, including patients with recurrent glioblastoma.

Epidiolex (GW Pharmaceuticals) investigational cannabidiol product for the treatment of Dravet syndrome.

Orphan drug designations

Cannibidiol (CBD) (Insys Therapeutics) for the treatment of Lennox-Gestaut Syndrome (LGS), a rare form of pediatric-onset epilepsy, as well as for the treatment of Druvet Syndrome.

EX404 (EffRx) for the treatment of pediatric polycystic ovary syndrome.

EPI-743 or vatiquinone (Edison) for treatment of Leigh syndrome.

MI-100 (Makindus) for the treatment of Stargardt's disease.

First-time generic approval

Testosterone gel (equiv to Testim)

Auxilium Pharmaceuticals