Skip to Main Content
Contribute Try STAT+ Today

Six advocacy groups have asked the federal government to sidestep a handful of patents on a pricey drug used to treat a rare form of muscular dystrophy as part of an ongoing campaign to provide wider access to high-cost medicines.

In a letter sent on Wednesday to the Department of Health and Human Services, the groups argued that the patents — which are either owned or licensed by Sarepta Therapeutics (SRPT) — failed to disclose federal funding for grants that were used to develop a drug known as Exondys 51 for treating Duchenne muscular dystrophy. This is an accompanying memo sent to HHS.

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


What is it?

STAT+ is STAT's premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond.

What's included?

  • Daily reporting and analysis
  • The most comprehensive industry coverage from a powerhouse team of reporters
  • Subscriber-only newsletters
  • Daily newsletters to brief you on the most important industry news of the day
  • STAT+ Conversations
  • Weekly opportunities to engage with our reporters and leading industry experts in live video conversations
  • Exclusive industry events
  • Premium access to subscriber-only networking events around the country
  • The best reporters in the industry
  • The most trusted and well-connected newsroom in the health care industry
  • And much more
  • Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr.
  • As a matter of healthcare ethics and public policy, the government should note that Sarepta, in its third quarter investor call, projected that they would generate $300M in revenues from Exondys 51 in 2018. The patient population or Exondys 51 is only 13% of the Duchenne population of only 2,000 – 3,000 people, or perhaps fewer than 300 people. Especially since Sarepta’s drug was developed from NIH funded research, this unconscionable pricing must be stopped.

  • President Trump, please make this very critical medicine affordable for anyone to purchase. Thank you.

  • In light of the pricing in this post, the fears reported earlier in the day of a tariff on Chinese raw materials causing rise in drug prices seem ….. overblown at a minimum and ludicrous as well.

Comments are closed.