If approved, this would be the first marketing authorization for elamipretide, a first-in-class mitochondria-targeted therapeutic, and the first FDA-approved therapy for Barth syndrome.

The new Prescription Drug User Fee Act target date is April 29, 2025. The Food and Drug Administration (FDA) has extended the review period for the New Drug Application (NDA) for elamipretide for ...

"We continue to work closely with the Agency as it completes its review of the elamipretide NDA and are actively preparing to support broad access to this therapy for individuals living with Barth ...

Follows Positive FDA Advisory Committee Meeting Vote Concluding that Elamipretide is Effective for the Treatment of Barth Syndrome – NEEDHAM, Mass., Jan. 23, 2025 /PRNewswire/ -- Stealth ...

The US Food and Drug Administration (FDA) has delayed its decision on Stealth BioTherapeutics’ new drug application (NDA) for elamipretide, a treatment for the ultra-rare disease Barth syndrome.

Three months ago, the FDA’s Cardiovascular and Renal Drugs Advisory Committee voted 10-6 in support of elamipretide becoming the first approved treatment for Barth syndrome, an X-linked genetic ...