- Catalyst Pharmaceuticals on Thursday revealed it had received a "refusal to file" letter from the FDA, dealing a significant blow to its drug for a rare degenerative disease. The FDA deemed Catalyst's new drug application (NDA) for firdapse to be insufficiently complete and requested further information.
- 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 NDA is controversial because firdapse is highly similar to an older, but unapproved, drug called 3,4-Dap which has been manufactured and supplied for free by New Jersey-based Jacobus Pharmaceuticals.
Jacobus Pharmaceuticals is also attempting to win approval from the FDA for its version of the drug. It has made 3,4-Dap for over 20 years but never submitted the drug for regulatory approval. Instead, the small company has given away the drug for free. Doctors who wish to prescribe 3,4-Dap have to fill out a substantial amount of paperwork, rather than just write a prescription. Patients also have to travel to New Jersey to get the drug.
An approval of Catalyst's firdapse would change that. Firdapse has been granted an orphan drug designation by the FDA, which would grant Catalyst seven years of marketing exclusivity if approved. Patients and doctors fear Catalyst would raise the price dramatically, following a script similar to the strategies used by Valeant and Turing Pharmaceuticals. For its part, Catalyst said it hasn't decided on a price yet, according to the New York Times.
While the "refusal to file" letter from the FDA sets Catalyst back, the letter did not comment on efficacy or safety of firdapse.. "We expect to work closely with the FDA over the coming weeks in an effort to resolve the open issues and tod define a path forward for a successful resubmission," said Catalyst CEO Patrick McEnany in a statement.
It is unclear what effect this may have on Jacobus' drug application. The FDA does not take price into consideration when approving a drug.