Posted by Michael Wonder on 28 Nov 2017
Stealth BioTherapeutics granted fast track designation for the treatment of Barth syndrome
27 November 2017 - Stealth BioTherapeutics today announced that the U.S. FDA has granted fast track designation for its lead candidate, elamipretide, for the treatment of Barth syndrome.
“Fast Track designation is an important milestone which will facilitate Stealth’s efforts to develop an effective treatment for the Barth syndrome patient community, for whom there are currently no FDA-approved therapies,” said Stealth Chief Executive Officer Reenie McCarthy. “We are committed to developing treatments for patients suffering from rare mitochondrial diseases such as Barth syndrome, and look forward to working closely with the FDA in addressing this critical unmet need.”
In July 2017, Stealth initiated TAZPOWER, a Phase 2/3, randomised, double-blind, placebo-controlled crossover study that will evaluate the effects of daily treatment with elamipretide in patients with genetically confirmed Barth syndrome. Top-line results from the study are expected in 2018.
