Rare disease drugmaker Sarepta Therapeutics has announced the completion of the sale of its Rare Pediatric Disease Priority Review Voucher (PRV) for $102 million.
The PRV was granted to Sarepta after the FDA’s accelerated approval of Elevidys, designed for the treatment of ambulatory pediatric patients between the ages of 4 and 5 who have Duchenne muscular dystrophy (DMD) and a confirmed mutation in the DMD gene.
The FDA's Rare Pediatric Disease Priority Review Voucher Program awards priority review vouchers to sponsors of product applications for rare pediatric diseases that meet specific criteria. The program aims to encourage the development of new drugs and biologics that prevent and treat rare pediatric diseases, and the vouchers can be used to prioritize the review of future marketing applications for different products that are transferable.
In the recent statement, Sarepta expressed its intentions to allocate the funds obtained from the sale of the priority review voucher towards its research and development initiatives.
Developed in collaboration with Roche, Elevidys facilitates the specific generation of functional parts of dystrophin, a protein crucial for DMD, genetic mutations affect the protein's structure, impairing its normal function and leading to various musculoskeletal symptoms. Although the disease is rare, it is life-threatening and affects around 1 in every 3,500-5,000 newborn boys annually.