Stealth BioTherapeutics Announces "Path Forward" Despite Disappointing Delay for Ultra-rare Barth Syndrome

Axe falls on Stealth Bio staff after FDA rejects lead drug

Stealth BioTherapeutics' filing for Barth syndrome therapy elamipretide has been turned down by the FDA, but there may be a way forward for the drug

US FDA asks Stealth BioTherapeutics to resubmit application for rare genetic condition therapy

(Reuters) -The U.S. Food and Drug Administration has asked for a resubmission of Stealth BioTherapeutics’ marketing application for its experimental therapy targeting a rare genetic condition, Barth syndrome, the company said on Thursday. The drug developer added that it has implemented a 30% workforce reduction to conserve resources for a potential resubmission. The federal health regulator’s decision marks another setback in Stealth’s prolonge…

A rare disease drug is rejected, even as the FDA talks about new approval pathway

The FDA withheld its endorsement of a drug for Barth syndrome, but decided it may be possible for Stealth BioTherapeutics to pursue accelerated approval.

Stealth BioTherapeutics Announces "Path Forward" Despite Disappointing Delay for Ultra-rare Barth Syndrome

Following five years of discussions and a positive advisory committee recommendation, FDA proposes a potential accelerated approval pathway requiring resubmission of the elamipretide NDABarth syndrome, a progressive, lethal, pediatric cardioskeletal disease often diagnosed at birth and with no approved therapies,…

FDA Rejects Stealth BioTherapeutics’ Barth Syndrome Treatment After 16.5-Month Review

The U.S. Food and Drug Administration (FDA) has decline […] The post FDA Rejects Stealth BioTherapeutics’ Barth Syndrome Treatment After 16.5-Month Review first appeared on GeneOnline News. The post FDA Rejects Stealth BioTherapeutics’ Barth Syndrome Treatment After 16.5-Month Review appeared first on GeneOnline News.

FDA rejects Stealth Bio’s Barth syndrome treatment after 16.5-month review - Pharmaceutical Technology

Stealth Bio has cut 30% of its workforce as it eyes a resubmission based on discussions with the FDA.