Despite FDA Concerns, Stealth BioTherapeutics Pursues Elamipretide Approval

Stealth BioTherapeutics is seeking FDA approval for elamipretide, for treatment of Barth syndrome, a rare genetic disease, even though the FDA has said it needs to see data from an additional phase 3 study.

Stealth acknowledged its risky approach but said that “neither the FDA nor the company has identified a feasible trial design due to the ultra-rare nature of this disease.”

In support of its new drug application for the peptide compound, the company cited the unmet need as there is no approved treatment for the condition characterized by cardiac abnormalities linked with heart failure and lowered life expectancy, recurrent infections, muscle weakness and delayed growth.

U.S. and EU regulators previously signaled their interest in elamipretide. The drug has received Rare Pediatric Disease, Fast Track and Orphan Drug designations from the FDA and Orphan Drug status from the EMA.