FDA Expedites Review of Stealth BioTherapeutics’ Elamipretide Drug After May Rejection, September Decision Date Set

The U.S. Food and Drug Administration (FDA) has agreed to expedite the review of Stealth BioTherapeutics' drug candidate elamipretide, intended to treat Barth syndrome, an ultra-rare disease. This decision follows the company's earlier rejection in May 2025 after its initial submission in January 2024. The FDA has set a planned decision date of September 26, 2025, signaling a potential shift in the agency's approach to rare disease therapies. Stealth BioTherapeutics had indicated it might have to cease operations without this regulatory progress. Meanwhile, other pharmaceutical industry developments include employers' concerns over health costs related to GLP-1 drugs, shifts in COVID-19 vaccine strategies, and legal and practical challenges facing direct-to-patient pharmaceutical sales. Additionally, the European Federation of Pharmaceutical Industries and Associations (EFPIA) is mobilizing efforts in the EU to protect innovative drugs following the confirmation of a U.S. tariff cap. The FDA is also addressing deadlines for drug distributors regarding electronic product tracking data exchange.