Sarepta Therapeutics was dealt a surprising setback Monday when the Food and Drug Administration rejected its marketing application for a second drug that aimed to treat children with Duchenne muscular dystrophy, a rare, inherited muscle-wasting disease.

In a statement, Sarepta said the FDA denied the approval of its drug, called Vyondys 53, due to the risk of infections related to intravenous infusion ports and kidney toxicity seen in animal experiments.

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

GET STARTED

What is it?

STAT Plus 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
  • Online intelligence briefings
  • Frequent opportunities to engage with veteran beat reporters and industry experts
  • 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.

Leave a Comment

Please enter your name.
Please enter a comment.

  • My two son is duchenne Muscular dystrophy (DMD) patient, error exon 53. 1-Ayush Tiwari 13y old. 2-Harsh Tiwari 8y old. I live in INDIA.

  • This is all due to hubris. Management should have OVER-invested in this trial since 2016. Not only did they fail to do that, they also failed to provide the follow up data FDA requested since Exondys 51 was approved almost 4 years ago. They have a very conditional approval for Exondys 51, and who knows what the implications are for that drug? They’ve put their entire franchise in jeopardy.

    This is a terrible outcome for patients and their families. Really tragic because this company, over the last four years, could have built a champion for rare diseases. Instead management is unlikely to be able to dig itself out of its pipeline hole.

    • The terrible outcome for patients and taxpayers happened when the FDA approved Exondys 51. There is a reason Sarepta hasn’t completed the required studies; the drug, very likely, doesn’t work. It never did.

    • My son has DMD and we were waiting for Vyondys 53 to be approved. Looks like it’s not going to happen anytime soon. Nitin, you could contact me if you would like to discuss.

  • ACER, Chiesi’s recent CRL on CF, the weird ZGNX pushback, the bizarre approval of Ruzurgi contra to the orphan drug act. There’s no doubt there is a policy change at the FDA, just not sure the FDA knows what it is yet

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

Privacy Policy