FDA Approves First Treatment for Rare Autoimmune Disorder

Catalyst Pharmaceuticals, Inc. received approval from the FDA for Firdapse (amifampridine) tablets for the treatment of Lambert-Eaton myasthenic syndrome (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. 
 
In people with LEMS, the body's own immune system attacks the neuromuscular junction (the connection between nerves and muscles) and disrupts the ability of nerve cells to send signals to muscle cells. The prevalence of LEMS is estimated to be three per million individuals worldwide. 
 
The efficacy of Firdapse was studied in two clinical trials that included 64 patients who received Firdapse or placebo. The studies measured the Quantitative Myasthenia Gravis score (a scale assessing muscle weakness) and the Subject Global Impression (a scale on which patients rated their overall impression of the effects of the study treatment on their physical well-being). For both measures, the patients receiving Firdapse experienced a greater benefit than those on placebo. 
 
The most common side effects experienced by patients in the clinical trials were burning or prickling sensation, upper respiratory tract infection, abdominal pain, nausea, diarrhea, headache, elevated liver enzymes, back pain, hypertension and muscle spasms.
 
The FDA granted this application Priority Review and Breakthrough Therapy designations. Firdapse also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

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