Stealth BioTherapeutics Inc.’s elamipretide moved a step closer to US approval with a Food and Drug Administration advisory committee’s positive vote that the drug is effective for the treatment of Barth syndrome.
Stealth’s Elamipretide Gets US FDA Panel Nod, But Not A Glowing Endorsement, For Barth Syndrome
The Cardiovascular and Renal Drugs Advisory Committee voted 10-6 that efficacy had been shown for the ultra-rare disease, but even panelists in the majority questioned whether the product satisfied the threshold requirement for an adequate and well-controlled study.
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