Catalyst's long-delayed Firdapse snags FDA nod, but pricing questions loom

It took a breakthrough designation, orphan drug status, an embarrassing up-front rejection letter and some extra trials, but Catalyst Pharmaceuticals has finally crossed the finish line with Firdapse.

The FDA approved the drug late Wednesday to treat Lambert-Eaton myasthenic syndrome (LEMS), a rare autoimmune disorder often characterized by weakness in the limb muscles. The company expects to roll out the new treatment—which BioMarin has marketed in Europe since 2009—in the first quarter of next year.

“We continue to prepare for what we hope will be a successful launch based on productive discussions with payers, our efforts in patient identification and our development of a field-based commercial infrastructure,” company CEO Patrick McEnany said in a statement.

RELATED: Catalyst Pharmaceuticals hit by FDA extra studies request for Firdapse

It’s been a long journey for the therapy, which back in 2016 received a Refuse to File letter from the FDA after the agency deemed one late-stage study “not sufficiently complete.” Two months later, regulators were back at Catalyst’s door asking for another trial and additional short-term toxicology studies.

Fast forward to Wednesday, and Coral Gables, Florida-based Catalyst was touting Firdapse as the “first evidence-based medicine approved to treat LEMS.” But it’s not the first medicine used to treat LEMS, and that’s where Catalyst could run into issues.

Patients have had access to doses of 3,4-Dap, a treatment similar to Firdapse, for more than 20 years, courtesy of Jacobus Pharmaceuticals. That drug has never nabbed an FDA approval and consequently can’t legally be marketed in the U.S., but it could make it tough for payers to swallow Firdapse’s price tag—especially if Catalyst opts for a lofty sticker typical of orphan-designated drugs.

For now, the company is keeping investors in the dark; it plans to reveal Firdapse’s price on a conference call next month. In the meantime, though, it’s talking up its Catalyst Pathways patient services program, which is “designed to assist patients and physicians with education, clinical diagnostic tools and assistance in navigating the reimbursement landscape,” McEnany said. He added that Catalyst has had “productive discussions with payers.”