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

 
• By Sue Sutter

The 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.

Climbing steep hill
Stealth faces a steep hill at the FDA cardio-renal adcomm on elamipretide. • Source: Shutterstock

Stealth BioTherapeutics Inc. faces an uphill climb at a 10 October advisory committee meeting given the US Food and Drug Administration staff’s view 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 the efficacy of Stealth Biotherapeutics’ elamipretide for treatment of Barth syndrome.

  • A randomized, placebo-controlled trial failed its primary endpoint, a long-term extension is uninterpretable because it was open-label, and a historical control comparison suffered from methodological issues, the FDA’s advisory committee briefing document states

A randomized, placebo-controlled, crossover trial failed its primary clinical endpoints, and an uncontrolled, open-label, long-term extension of that study is uninterpretable for efficacy, the FDA said in a briefing

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