FDA approves Catalyst’s Firdapse

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FDA approves Catalyst’s Firdapse

By Sandra Levy - 12/07/2018
Catalyst Pharmaceuticals has received the ok from the Food and Drug Administration for Firdapse (amifampridine) tablets for the treatment of Lambert-Eaton myasthenic syndrome, or LEMS in adults. LEMS is a rare autoimmune disorder that affects the connection between nerves and muscles and causes weakness and other symptoms in affected patients. This is the first FDA approval of a treatment for LEMS.

“There has been a long-standing need for a treatment for this rare disorder,” director of the division of neurology products in the FDA’s Center for Drug Evaluation and Research Billy Dunn said. “Patients with LEMS have significant weakness and fatigue that can often cause great difficulties with daily activities.”

In people with LEMS, the body’s own immune system attacks the neuromuscular junction, which is the connection between nerves and muscles, and disrupts the ability of nerve cells to send signals to muscle cells. LEMS may be associated with other autoimmune diseases, but more commonly occurs in patients with cancer such as small cell lung cancer, where its onset precedes or coincides with the diagnosis of cancer. The prevalence of LEMS is estimated to be three per million individuals worldwide.

The FDA granted this application Priority Review and Breakthrough Therapy designations. Firdapse also received the Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

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