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

More from Rare Diseases

More from US Advisory Committees