Stealth BioTherapeutics Granted Fast Track Designation for the Treatment of Barth Syndrome

Stealth BioTherapeutics (Stealth) announced that the U.S. Food and Drug Administration (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, randomized, 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.

Barth syndrome is a rare genetic mitochondrial disease, caused by mutations in the TAZ gene.  The disease is characterized by skeletal muscle weakness, cardiac abnormalities often leading to heart failure, recurrent infections, delayed growth and reduced life expectancy. There are currently no FDA-approved therapies for the disease.

Elamipretide was previously granted Fast Track designation for the treatment of primary mitochondrial myopathy (PMM), another rare mitochondrial disease, in December 2015. The FDA’s Fast Track program facilitates the development and review of drugs to treat serious conditions with unmet medical needs. The designation provides the opportunity for more frequent meetings with the FDA over the course of development, and allows a drug company to submit individual sections of its New Drug Application (NDA) for review as they are ready, rather than waiting until all sections of the NDA are complete. Further, the designation also increases the likelihood of eligibility for priority review and accelerated approval if relevant criteria are met.