- Safety & Recalls
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FDA Pipeline Preview-September 2013
Priority review, breakthrough therapy, fast-track designations, orphan drug designations, first-time generic approval
Recommended for approval
â¾ Riociguat (Bayer AG) oral soluble guanylate cyclase (sGC) stimulator for the treatment of 2 forms of pulmonary hypertension: pulmonary arterial hypertension of WHO Group 1 and chronic thromboembolic pulmonary hypertension of WHO Group 4.
Priority review
â¾ Sorafenib (Bayer AG and Onyx Pharmaceuticals) supplemental New Drug Application for the treatment of locally advanced or metastatic radioactive iodine-refractory differentiated thyroid cancer.
Breakthrough therapy
designation
â¾ Bimagrumab (Novartis) for the treatment of patients with sporadic inclusion body myositis.
â¾ Amifampridine phosphate (Firdapse, Catalyst Pharmaceutical) for the symptomatic treatment of patients with Lambert-Eaton Myasthenic Syndrome (LEMS).
Fast-track designation
â¾ SGR-MD-02 (Galectin Therapeutics) for the treatment of fatty liver disease with advanced fibrosis.
Orphan drug designations
â¾ E7777 (Eisai) for the treatment of cutaneous T-cell lymphoma.
â¾ VS-6063 (defactinib, Verastem) cancer stem cell inhibitor for the treatment of mesothelioma.
â¾ GR-MD-O2, galactoarabino-rhamnogalacturonate, for the treatment of nonalcoholic steatohepatitis with hepatic fibrosis, commonly known as fatty liver disease with advanced fibrosis.
â¾ MM-111 (Merrimack Pharmaceuticals Inc. [MACK]) for the treatment of esophageal, gastric, and gastroesophageal junction cancers.
â¾ ALN-AT3 (Alnylam), a subcutaneous antithrombin for rare bleeding disorders, for the treatment of hemophilia A and for hemophilia B.
â¾ First-time
generic
approval
Ranolazine extended-release tablets in the 500-mg and 1,000-mg strengths (equiv to Ranexa)
lupin
FDA Approves Pfizer’s Gene Therapy for Beqvez for Hemophilia
April 26th 2024Beqvez (fidanacogene elaparvovec) is priced at $3.5 million, which is on parity with Hemgenix, the first one-time therapy to treat adults with hemophilia B. Pfizer’s warranty will refund insurers and continue to provide coverage for patients if they change insurers.
FDA Issues Complete Response Letter for Pz-Cel to Treat Epidermolysis Bullosa
April 22nd 2024Prademagene zamikeracel is a cell therapy designed to incorporate the functional collagen-producing COL7A1 gene into a patient’s own skin cells. The FDA is asking for additional information on manufacturing practices.
