A new controversy is emerging over a costly treatment for Duchenne muscular dystrophy, a rare disease that can send boys to an early death, thanks to a new analysis suggesting that clinical data is so limited that it is impossible to determine whether the medication is worth a price tag of $300,000 or more.

The medicine, which is called Exondys 51 and marketed by Sarepta Therapeutics (SRPT), caused a heated public debate over regulatory approval standards and satisfying unmet medical needs during the run-up to its endorsement nearly three years ago by the Food and Drug Administration.

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

GET STARTED

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.

  • How dare ICER use the stated list price – ““many factors were ignored including mandatory discounts and rebates to insurers and pharmacy benefit companies.” This reads like another proof that this drug should never have been approved in the first place.

Sign up for our Daily Recap newsletter

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

Privacy Policy