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Stealth faces another hurdle as FDA delays decision on Barth syndrome drug
The FDA rejected the company’s initial NDA in 2021, citing the lack of an adequate trial to demonstrate efficacy.
thecardiologyadvisor.com2d
FDA Extends Review of Elamipretide for Barth Syndrome
The FDA has extended the review period for the New Drug Application for elamipretide for the treatment of Barth syndrome.
FierceBiotech7d
FDA delays decision on Stealth's 2nd shot at approval for ultrarare disease med
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 ...
Morningstar7d
Stealth BioTherapeutics Announces PDUFA Action Date Extension for Elamipretide to Treat Patients with Barth Syndrome
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.
Stockhouse3mon
Stealth BioTherapeutics Announces PDUFA Action Date Extension for Elamipretide to Treat Patients with Barth Syndrome
"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 ...