The FDA rejected the company’s initial NDA in 2021, citing the lack of an adequate trial to demonstrate efficacy.

An FDA advisory committee may have ruled in elamipretide’s favor back in October, but it looks like Stealth BioTherapeutics ...

The FDA has extended the review period for the New Drug Application for elamipretide for the treatment of Barth syndrome.

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 ...

Stealth BioTherapeutics Inc. (the "Company" or "Stealth"), a clinical-stage biotechnology company focused on the discovery, ...

A US Food and Drug Administration (FDA) advisory committee (AdCom) has voted in support of Stealth Biotherapeutics’ ...

Barth syndrome (BTHS) is one of the rare X linked recessive diseases that appear in infancy with a triad of myocardial and skeletal muscle diseases, neutropenia and growth retardation. The pathogenic ...

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.

Barth syndrome (BTHS) is an X linked recessive disorder caused by a mutation in the tafazzin (TAZ) gene classically associated with the triad of neutropaenia and cardiac and skeletal myopathies. Here ...

The US Food and Drug Administration (FDA) has delayed its decision on Stealth BioTherapeutics’ new drug application (NDA) for elamipretide, a treatment for the ultra-rare disease 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.

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 ...