1. Pink Sheet
  2. >>Product Reviews

With Sarepta’s Vyondys, US FDA Staff Saw Opportunity To Right Perceived Wrongs With Exondys

 
• By Sue Sutter

ODE I director Ellis Unger chafed at the notion of approving the Duchenne muscular dystrophy drug golodirsen on the same unvalidated surrogate marker, this time in the face of infection and renal toxicity concerns; OND’s Peter Stein granted Sarepta’s appeal while also calling attention to the company’s failure to timely conduct the eteplirsen confirmatory trial.

Drug Review Profile regular column
Sarepta successfully appealed a complete response letter for Vyondys 53.

For some at the US Food and Drug Administration, the review of Sarepta Therapeutics Inc.’s Duchenne muscular dystrophy drug Vyondys 53 (golodirsen) provided an opportunity to try to right the wrongs they saw with the accelerated approval of Exondys 51 (eteplirsen) three years earlier.

The mere fact of eteplirsen’s approval, and the precedent it set, was wrong in the eyes of some agency staffers

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Pink Sheet for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required
Start Free Trial

Already a subscriber?

More from Drug Review Profiles

More from Product Reviews

FDA Adcomms Are Back: Four Cancer Drugs, COVID-19 Vaccine Formulations To Get Reviews

 
• By Sue Sutter and Bridget Silverman

The Oncologic Drugs Advisory Committee will meet for two days in mid-May, followed by a Vaccines and Related Biological Products Advisory Committee’s review of the 2025-2026 COVID-19 vaccine formulation.

Unlocking Opportunities: How To Engage With The EMA On Animal Testing Alternatives

 
• By Neena Brizmohun

The European Medicines Agency, like its counterpart in the US, is increasingly focusing on the use of alternatives to animal testing.

Opioid Safety: US FDA Hears Mixed Messages On Adding Posmarketing Studies To Labeling

 
• By Sue Sutter

Some advisory committee members said adding quantitative data on misuse, abuse, opioid use disorder and overdose to labeling would be helpful, but others worried the two epidemiological studies were not sufficiently generalizable to a broader population.