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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.com7h
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.
FierceBiotech5d
FDA delays decision on Stealth's 2nd shot at approval for ultrarare disease med
An FDA advisory committee may have ruled in elamipretide’s favor back in October, but it looks like Stealth BioTherapeutics ...
ENDPOINTS NEWS5d
FDA de­lays de­ci­sion on Stealth’s Barth syn­drome drug; Tar­getRx rais­es $50M
FDA delays Stealth BioTherapeutics' elamipretide decision to April 29; Valneva's Ixchiq succeeds in child safety trial; TargetRx raises $50M; Ab&B Bio-Tech files for HKEX IPO ...
Healio9y
Barth's Syndrome
Barth’s syndrome in an inherited disorder in an X-linked fashion. The cardiac component includes dilated cardiomyopathy which can at times cause severe systolic congestive heart failure.
Stealth BioTherapeutics Announces PDUFA Action Date Extension for Elamipretide to Treat Patients with Barth Syndrome
Stealth BioTherapeutics Announces PDUFA Action Date Extension for Elamipretide to Treat Patients with Barth Syndrome ...
The Pharma Letter4d
FDA delays PDUFA action date for elamipretide
US clinical-stage drug developer Stealth BioTherapeutics announced that the US Food and Drug Administration (FDA) has ...
Monthly Prescribing Reference5d
FDA Extends Review of Elamipretide 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 ...
Morningstar5d
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.