FDA misses decision deadline on Stealth’s Barth syndrome drug

US biotech firm Stealth BioTherapeutics (OTCQB: MITO) is facing renewed uncertainty after the American medicines regulator failed to deliver a decision on the company’s new drug application for elamipretide, its potential treatment for Barth syndrome, by the expected April 29 deadline.

Stealth filed for approval of elamipretide in January 2024, with the Food and Drug Administration (FDA) later granting a priority review and confirming post-marketing plans. While a positive advisory committee vote followed in October, the outcome was not unanimous, with 6 voting against and 10 in favor. The agency subsequently requested further analyses, delaying the original January decision date.

Stealth chief executive Reenie McCarthy said discussions over labeling have now begun and indicated the company is waiting to hear when a revised decision can be expected. She noted that these details would help determine how the company continues to offer the therapy through emergency or expanded access while awaiting a final regulatory call.