FDA Grants Elamipretide Orphan Drug Designation for Duchenne Muscular Dystrophy and Friedreich Ataxia

The Food and Drug Administration (FDA) has granted elamipretide (Bendavia; Stealth BioTherapeutics, Cayman Islands) orphan drug designation for Duchenne muscular dystrophy (DMD) in addition to Friedreich ataxia. In a phase 2a trial (NCT05168774), currently enrolling participants, elamipretide will be evaluated safety, visual function, and cardiac function for Friedreich ataxia (and DMD) treatment.

"We are pleased that the FDA has recognized the high unmet need for innovative treatments for Friedreich ataxia and DMD," said chief executive officer Reenie McCarthy. "We are delighted to be working closely with Dr. Lynch and the Children's Hospital of Pennsylvania to evaluate elamipretide as a potential treatment for the progressive visual dysfunction and cardiomyopathy that affects visual quality of life and lifespan for individuals affected with Friedreich ataxia."

Elamipretide has been shown to improve frataxin levels in Friedreich ataxia patient-derived cells. Data demonstrating that administration of elamipretide in combination with a phosphorodiamidate morpholino oligomer (PMO) significantly improves dystrophin expression levels in the X-linked muscular dystrophy (mdx) mouse model.  

McCarthy continued, "We look forward to further discussions with the FDA regarding our development initiatives, which we hope will bring new options to patients suffering from these devastating diseases."