RARE Daily

Sarepta Sells Priority Review Voucher for $102 Million

July 5, 2023

Rare Daily Staff

Sarepta Therapeutics sold a Rare Pediatric Disease Priority Review Voucher that it was awarded following U.S. Food and Drug Administration accelerated approval of Elevidys.

The gene therapy was approved for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene.

DMD is caused by a change or mutation in the gene that encodes instructions for dystrophin. Symptoms of DMD usually appear in infants and toddlers. Elevidys addresses the root genetic cause of Duchenne by delivering a gene that codes for a shortened form of dystrophin to muscle cells known as Elevidys micro-dystrophin. It is a single-dose gene transfer therapy.

Under the Rare Pediatric Disease Priority Review Voucher (PRV) Program, FDA awards priority review vouchers to sponsors of rare pediatric disease product applications that meet certain criteria. The program is intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. PRVs can be redeemed to receive priority review of a subsequent marketing application for a different product, sold or transferred.

Sarepta will invest proceeds from the sale of the PRV into our R&D efforts to support the development of more transformative therapies.

Photo: Doug Ingram, president and CEO of Sarepta


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