Source – Sarepta Therapeutics
On July 5, 2023, Sarepta Therapeutics, a leading company in precision genetic medicine for rare diseases, successfully sold its Rare Pediatric Disease Priority Review Voucher (PRV), receiving a payment of $102 million. The PRV was granted to Sarepta after the accelerated approval of their drug ELEVIDYS for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the DMD gene.
The Rare Pediatric Disease Priority Review Voucher Program, administered by the US Food and Drug Administration (FDA), awards priority review vouchers to sponsors of rare pediatric disease product applications that meet specific criteria. This program aims to incentivize the development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. These vouchers can be used to expedite the review process for future marketing applications of different products or be sold or transferred.
Sarepta plans to reinvest the proceeds from the PRV sale into their research and development efforts to further support the advancement of transformative therapies. This strategic allocation of funds will enable Sarepta to continue their mission of developing innovative treatments for rare diseases, benefiting patients in need.
