thecardiologyadvisor.com1d
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.
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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.
FierceBiotech6d
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 ...
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 ...
GlobalData on MSN3mon
FDA AdCom votes in favour of Stealth’s Barth syndrome drug
A US Food and Drug Administration (FDA) advisory committee (AdCom) has voted in support of Stealth Biotherapeutics’ ...
Nature6d
Barth Syndrome and Mitochondrial Cardiolipin Metabolism
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 ...