Elamipretide is in the news again! FDA has acknowledged the urgent, unmet need of the Barth syndrome community and granted elamipretide a Priority Review designation after initially assigning a standard review designation. If elamipretide is approved by FDA, Stealth BioTherapeutics may be granted a Priority Review Voucher. This designation update does not change the anticipated timing of the FDA review process but may help catalyze future drug development for Barth syndrome. 

Why do we care? The Priority Review Voucher serves as a potent incentive for biotech and pharmaceutical companies to invest in drug development for rare and ultra-rare diseases. Due to small patient populations, the research and development costs for most rare and ultra-rare indications will exceed the anticipated profits and preclude investment in these areas despite high need. The Priority Review Voucher thus serves as a catalyst for innovation and investment in rare disease.  

Read the press release here. 

Stay Tuned: Once plans are solidified, the Barth Syndrome Foundation will coordinate a Town Hall to define the role of the Barth syndrome community in the upcoming Advisory Committee or AdComm meeting.