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Stealth’s Elamipretide: Is A New Preapproval Trial Feasible?

 
• By Sue Sutter

Members of the Cardiovascular and Renal Drugs Advisory Committee repeatedly challenged Stealth’s assertion that a new randomized trial in Barth syndrome was not possible, but also said potential functional unblinding and other challenges could compromise a new study.

Trash out the window
One adcomm member said approving elamipretide now would be throwing away the opportunity for future studies. • Source: Shutterstock

A US Food and Drug Administration advisory committee review of Stealth BioTherapeutics Inc.’s elamipretide repeatedly circled back to the question of whether further preapproval data collection would be feasible as well as informative.

Members of the Cardiovascular and Renal Drugs Advisory Committee were split on this issue, just as they were divided on whether the drug’s efficacy

Key Takeaways

  • Stealth said it did not attempt a new randomized trial of elamipretide because it could not agree with the FDA on a design.

  • The FDA said Stealth was reluctant to undertake another withdrawal study due to uncertainty about when symptoms would recur in a washout period

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