US FDA grants fast-track status to Stealth’s elamipretide

The US Food and Drug Administration (FDA) has granted fast-track designation for Stealth BioTherapeutics’ lead candidate, elamipretide, to treat patients with Barth syndrome.

Barth syndrome is a rare genetic mitochondrial disease caused by mutations in the Tafazzin (TAZ) gene and results in skeletal muscle weakness, cardiac abnormalities that often lead to heart failure, recurrent infections, delayed growth and reduced life expectancy.

There are currently no FDA-approved therapies available for the disease.

Stealth BioTherapeutics CEO Reenie McCarthy said: "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.

"Fast-track designation is an important milestone which will facilitate Stealth’s efforts to develop an effective treatment for the Barth syndrome patient community."

"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."

The designation supports the development and review of new therapies that can be potentially used to treat serious conditions with unmet medical needs.

In July this year, the biopharmaceutical company initiated a randomised, double-blind, placebo-controlled, crossover Phase II / III TAZPOWER study that would evaluate the effects of daily use of elamipretide in patients with genetically confirmed Barth syndrome.

Top-line results from the study are expected next year.

In December 2015, Stealth’s elamipretide was granted fast-track status for the treatment of primary mitochondrial myopathy (PMM), another form of rare mitochondrial disease.