51. Firdapse

Prescription and pills

Active ingredient: amifampridine
Disease: Lambert-Eaton myasthenic syndrome
Peak sales estimate: $364 million in 2024
Approved: Nov. 28
Company: Catalyst Pharmaceuticals

The scoop: Catalyst Pharmaceuticals' Firdapse didn’t have the easiest path to approval. Back in 2016, the FDA handed it a Refuse to File letter after deeming one late-stage study “not sufficiently complete.” Regulators followed up two months later with requests for another trial and short-term toxicology studies. Fast forward to this November, though, and Catalyst had pinned down the FDA’s first approval for rare autoimmune disorder Lambert-Eaton myasthenic syndrome. Turns out, though, snagging the OK may not have been the hardest part. Firdapse will go up against a similar treatment in the market that isn't FDA approved but has been accessible for 20 years, and that competition could make Firdapse’s $375,000 price tag hard for payers to swallow. — Carly Helfand

51. Firdapse

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