GlobalData on MSN3d
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.
Stealth BioTherapeutics Announces PDUFA Action Date Extension for Elamipretide to Treat Patients with Barth Syndrome
Stealth BioTherapeutics Inc. (the "Company" or "Stealth"), a clinical-stage biotechnology company focused on the discovery, ...
Monthly Prescribing Reference4d
FDA Extends Review of Elamipretide for Barth Syndrome
The Food and Drug Administration (FDA) has extended the review period for the New Drug Application (NDA) for elamipretide for the treatment of Barth syndrome, a rare genetic disorder.
FierceBiotech4d
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 ...
Morningstar4d
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.
Nature3d
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 ...
The Pharma Letter3d
FDA delays PDUFA action date for elamipretide
US clinical-stage drug developer Stealth BioTherapeutics announced that the US Food and Drug Administration (FDA) has ...
ENDPOINTS NEWS4d
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 ...