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Stealth BioTherapeutics’ Rare Genetic Disease Treatment Gains Fast Track

There are currently no other FDA-approved treatments for Barth syndrome

By: Betsy Louda

, Contract Pharma

The U.S. Food and Drug Administration has granted Fast Track designation to Stealth BioTherapeutics‘ Elamipretide, a treatment for 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’s 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.”

 

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. 

 

 

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