BOSTON (PRWEB)
August 25, 2021

The Barth Syndrome Foundation (BSF) expresses immense gratitude to Stealth BioTherapeutics (Stealth) for submitting the new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for the potential use of elamipretide as a treatment for those living with Barth syndrome.

Petitioned by Barth syndrome families and friends in 2020, BSF and clinical key opinion leaders urgently appealed to both Stealth and the FDA to submit and approve, respectively, the NDA for elamipretide based on data from clinical trials. Stealth’s submission of the NDA is a critical step forward for the entire Barth syndrome community. Importantly, data support the drug’s safety, as well as efficacy in most patients. Barth syndrome patients and their families have met with FDA to express their willingness to accept some risk that the treatment might not work for all patients. Concern persists, though, that FDA may not file the NDA and thus not initiate a full review. Such a decision would be devastating to the Barth syndrome community who has resoundingly communicated a willingness to accept some uncertainty of benefit to have access to a drug that could potentially offer a treatment option.

Barth syndrome is a life-threatening and life-limiting genetic disease and presently one of the more than 6,500 rare diseases without an approved treatment. If approved, elamipretide would be the first-ever approved treatment for use in Barth syndrome.

BSF will continue to support regulatory efforts for approval – exhausting all avenues available to ensure patients’ voices are actively considered – and push for policies that acknowledge the inordinate challenges of drug development in ultra-rare diseases.

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