Reeling from an unexpected move by regulators, Catalyst Pharmaceuticals (CPRX) is threatening to hit back.

Until recently, the drug maker had been riding high. Last November, the Food and Drug Administration approved its medicine for treating adults with a rare neuromuscular disorder called LEMS, which meant Catalyst has seven years of marketing exclusivity. And despite controversy over its $375,000 price tag, the company appeared to have silenced some critics with assistance programs that kept patient out-of-pocket costs low. Meanwhile, its soaring stock price pleased investors.

Unlock this article by subscribing to STAT Plus and enjoy your first 30 days free!


What is it?

STAT Plus is a premium subscription that delivers daily market-moving biopharma coverage and in-depth science reporting from a team with decades of industry experience.

What's included?

  • Authoritative biopharma coverage and analysis, interviews with industry pioneers, policy analysis, and first looks at cutting edge laboratories and early stage research
  • Subscriber-only networking events and panel discussions across the country
  • Monthly subscriber-only live chats with our reporters and experts in the field
  • Discounted tickets to industry events and early-bird access to industry reports

Leave a Comment

Please enter your name.
Please enter a comment.

  • Hmmm, the potential for ‘off-label use.’ Is there anyone who actually believes that Catalyst wasn’t counting on the potential for that with their drug in the first place?
    [And as Catalyst ponders/ threatens, one might observe that their projection of a 10x larger market size was a questionable bit of bait [ not fraud, of course] for drawing investors? One might … ]

Sign up for our Daily Recap newsletter

A roundup of STAT’s top stories of the day in science and medicine

Privacy Policy