Catalyst Pharmaceuticals, Inc., a biopharmaceutical company focused on therapies for people with rare debilitating, chronic neuromuscular, and neurological diseases, has received U.S. Food and Drug Administration (FDA) approval for Firdapse (amifampridine) 10 mg tablets to treat adults with Lambert-Eaton myasthenic syndrome (LEMS).
Firdapse is expected to be commercially available early in the first quarter of 2019.
LEMS is a rare autoimmune disease that affects approximately one in 100,000 people in the United States. The most common symptoms of LEMS are proximal muscle weakness and fatigue. Symptoms can be life threatening when the weakness involves respiratory muscles. Approximately 50 percent of LEMS patients have an underlying malignancy, typically small cell lung cancer.
The submission of the Firdapse NDA came following positive results from two Phase 3 studies where patients treated with Firdapse experienced rapid, significant, and sustained improvements in muscle function, and reduced weakness and fatigability compared to patients receiving placebo. The drug previously had received Orphan Drug designation and Breakthrough Therapy designation, as well as Priority Review from the FDA. Firdapse is the first and only approved drug in Europe for treatment of LEMS.
Gary Ingenito, M.D., Ph.D., chief medical officer at Catalyst, said the company plans to continue to work with the FDA to evaluate other potential indications for Firdapse.
(Source: Catalyst Pharmaceuticals, Inc.)