FDA Grants Priority Voucher for Rare Pediatric Disease Treatment Drug
Published Date: 12/19/2025
Notice
Summary
The FDA just gave Stealth BioTherapeutics a special priority review voucher for their rare pediatric disease drug, FORZINITY (elamipretide), approved in September 2025. This voucher speeds up future drug reviews, helping get important medicines to kids faster. It’s a big win for patients with Barth syndrome and could save time and money in drug development.
Analyzed Economic Effects
2 provisions identified: 2 benefits, 0 costs, 0 mixed.
Priority Review Voucher Issued for FORZINITY
The FDA issued a priority review voucher to Stealth BioTherapeutics for FORZINITY (elamipretide), which the agency approved on September 19, 2025. FORZINITY is indicated to improve muscle strength in adult and pediatric patients with Barth syndrome weighing at least 30 kg, and the voucher is intended to speed future drug reviews for rare pediatric disease products.
Voucher Lowers Development Time and Cost for Sponsor
The priority review voucher granted to the sponsor (Stealth BioTherapeutics) can shorten the review time for a future drug application and thus may save time and money in drug development for the sponsoring company.
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