- Catalyst Pharmaceuticals said the Food and Drug Administration has asked for an additional clinical study on the company's lead drug candidate, a little over two months after the regulator found an initial drug application insufficient.
- Shares of Catalyst dropped by nearly half after Tuesday's announcement, which lengthened firdapse's path to approval. The FDA indicated it could accept a small, short-term study in addition to several toxicology studies as part of the drug's application, the company said.
- Firdapse is designed to treat Lambert-Eaton Myasthenic syndrome (LEMS), a potentially fatal autoimmune disease which leads to the progressive muscle weakening. Catalyst estimates there are currently 3,000 LEMS patients in the U.S.
Catalyst's application for approval of firdapse is controversial because the drug's similarity to another LEMS treatment which has been provided to patients for free by Jacobus Pharmaceuticals.
Although Jacobus has made the drug, known as 3,4-Dap for over 20 years, it hadn't sought regulatory approval until recently. The FDA granted Catalyst's firdapse an orphan drug designation, making the drug eligible for seven years of market exclusivity and effectively shutting out Jacobus if approved.
The FDA's requirement of additional studies for firdapse could strain Catalyst's financials. The company indicated it had $52 million in cash and investments as of March 31 but "believes that it has the cash resources required to complete the additional studies."
Firdapse has already received regulatory approval in Europe for adults with LEMS and Catalyst is also developing the drug for treatment of congenital myasthenic syndrome.