FDA Pipeline Preview-September 2013


Priority review, breakthrough therapy, fast-track designations, orphan drug designations, first-time generic approval

Recommended for approval

◾ 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

◾ 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

◾ Bimagrumab (Novartis) for the treatment of patients with sporadic inclusion body myositis.

◾ Amifampridine phosphate (Firdapse, Catalyst Pharmaceutical) for the symptomatic treatment of patients with Lambert-Eaton Myasthenic Syndrome (LEMS).

Fast-track designation

◾ SGR-MD-02 (Galectin Therapeutics) for the treatment of fatty liver disease with advanced fibrosis.

Orphan drug designations

◾ E7777 (Eisai) for the treatment of cutaneous T-cell lymphoma.

◾ VS-6063 (defactinib, Verastem) cancer stem cell inhibitor for the treatment of mesothelioma.

◾ GR-MD-O2, galactoarabino-rhamnogalacturonate, for the treatment of nonalcoholic steatohepatitis with hepatic fibrosis, commonly known as fatty liver disease with advanced fibrosis.

◾ MM-111 (Merrimack Pharmaceuticals Inc. [MACK]) for the treatment of esophageal, gastric, and gastroesophageal junction cancers.

◾ ALN-AT3 (Alnylam), a subcutaneous antithrombin for rare bleeding disorders, for the treatment of hemophilia A and for hemophilia B.

◾ First-time

Ranolazine extended-release tablets in the 500-mg and 1,000-mg strengths (equiv to Ranexa)

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