FDA Grants Rare Pediatric Disease Designation to Elamipretide for Barth Syndrome

Stealth BioTherapeutics announced the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation for elamipretide for the treatment of Barth syndrome, an ultra-rare genetic condition.

Under the FDA's RPD designation program, the FDA may grant a priority review voucher to a sponsor who receives approval for a "rare pediatric disease," a serious and life-threatening disease that primarily affects individuals aged from birth to 18 years and fewer than 200,000 people in the United States. Upon FDA approval of elamipretide for Barth syndrome, Stealth BioTherapeutics is eligible for a voucher that can be used to obtain priority review for a subsequent human drug application upon meeting relevant statutory requirements associated with the RPD program.

"This designation for elamipretide underscores our alignment with the FDA around the significant and urgent unmet medical need for Barth patients," said Reenie McCarthy, Chief Executive Officer of Stealth.  "We hope to improve the healthspan of Barth patients, for whom cardiomyopathy, exercise intolerance and debilitating fatigue limit life expectancy and impair quality of life.  We are also expanding our development efforts to consider trials in patients suffering from overlapping metabolic cardiomyopathies in diseases such as Duchenne and Becker muscular dystrophies, and Friedreich's ataxia."

Barth syndrome is an ultra-rare genetic condition characterized by cardiac abnormalities often leading to heart failure and reduced life expectancy, recurrent infections, muscle weakness and delayed growth. Barth syndrome occurs almost exclusively in males and is estimated to affect one in 200,000 to 400,000 individuals worldwide. There are currently no FDA- or EMA-approved therapies for patients with Barth syndrome.

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