Stealth’s Elamipretide: Efficacy In Barth Syndrome Not Shown, US FDA Says

 
• By Sue Sutter
  
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Cardiovascular and Renal Drugs Advisory Committee will consider whether open-label extension data from a randomized trial that failed its primary endpoint, along with a historical control comparison, are enough to support approval in the ultra-rare disease.

Man climbing mountain.
Stealth faces a steep hill at the FDA cardio-renal adcomm on elamipretide. (Shutterstock)

Stealth Biotherapeutics faces an uphill climb at a 10 October advisory committee meeting given US Food and Drug Administration staff views’ that the available evidence does not establish elamipretide’s efficacy for treatment of Barth syndrome.

Key Takeaways

  • The FDA says available evidence does not establish efficacy of Stealth Biotherapeutics’ elamipretide for treatment of Barth syndrome.

A randomized, placebo-controlled, crossover trial failed its primary clinical endpoints, and an uncontrolled, open-label, long-term extension of that study is...

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