Boston, June 15, 2022 - Stealth BioTherapeutics (Stealth) announced yesterday that the U.S. Food and Drug Administration (FDA) has granted the company a meeting to discuss a possible new drug application (NDA) for elamipretide as a potential treatment for Barth syndrome. The company intends to present new data collected during the Open Label Extension (OLE) period.
In October 2021, Stealth received a refusal-to-file letter from the FDA for the same proposed treatment in Barth syndrome. At present, there are no FDA-approved therapies for the treatment of Barth syndrome.
“With other potential treatments years away from the clinic, people in our community are in dire need of a safe, effective therapy that can potentially attenuate the devastating clinical outcomes and implications to quality of life for people living with this debilitating and life-threatening disease,” said Emily Milligan, Executive Director of Barth Syndrome Foundation (BSF). “It is our hope that the FDA will review all available data this time that supports the approval of elamipretide as a potential therapy for Barth syndrome.”