Catalyst Pharma's Firdapse bet on congenital myasthenic syndromes turns sour
Months after Firdapse maker Catalyst Pharmaceuticals filed a lawsuit against the FDA, the Florida company on Wednesday said the drug had failed a pivotal trial in patients afflicted with congenital myasthenic syndromes (CMS), an umbrella term for rare neuromuscular disorders comprising a spectrum of more than 50 genetic defects.
The drug, known chemically as amifampridine, was sanctioned for use in adult Lambert-Eaton myasthenic syndrome (LEMS) patients in November 2018. The company is working on expanding the label to include patients with CMS, MuSK-positive myasthenia gravis (MuSK-MG) and spinal muscular atrophy (SMA).
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