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Stealth faces another hurdle as FDA delays decision on Barth syndrome drugThe FDA rejected the company’s initial NDA in 2021, citing the lack of an adequate trial to demonstrate efficacy.
The FDA has extended the review period for the New Drug Application for elamipretide for the treatment of Barth syndrome.
An FDA advisory committee may have ruled in elamipretide’s favor back in October, but it looks like Stealth BioTherapeutics will have to ...
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 ...
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.
Barth syndrome (BTHS) is a rare genetic disorder primarily affecting males, characterized by a range of symptoms including cardiomyopathy, skeletal myopathy, and neutropenia. The condition is ...
US clinical-stage drug developer Stealth BioTherapeutics announced that the US Food and Drug Administration (FDA) has ...
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 ...
Another X-linked form of cardiomyopathy, Barth syndrome, was initially described as X-linked cardioskeletal myopathy with abnormal mitochondria and neutropenia (low white blood count). This disorder ...
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