Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the groundbreaking leader in precision genetic medicine for rare diseases, has successfully sold its Rare Pediatric Disease Priority Review Voucher (PRV) for a massive $102 million. This PRV was a result of FDA accelerated approval of ELEVIDYS (delandistrogene moxeparvovec-rokl) for treating 4 to 5 year-old ambulatory pediatric patients with Duchenne muscular dystrophy (DMD) and a confirmed mutation in the DMD gene. It’s a groundbreaking event for rare diseases and a major victory for the rare disease community.
The FDA’s Rare Pediatric Disease Priority Review Voucher Program is designed to incentivize the development of new therapeutics for rare pediatric diseases. To that end, sponsors of successful applications for rare pediatric disease products are rewarded with Priority Review Vouchers. These vouchers can be redeemed to receive expedited review of a future application. Sarepta is proud to be a beneficiary of this program and intends to reinvest the proceeds of a PRV sale into research and development to help develop even more groundbreaking treatments for rare pediatric diseases.
About Sarepta Therapeutics
Sarepta is on an incredible mission to revolutionize precision genetic medicine and give hope to individuals with rare diseases. We are committed to tackling Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), while developing over 40 programs using the highly advanced Precision Genetic Medicine Engine. Our intention is to create accessible treatments which will have far-reaching, life-changing effects—bringing brighter futures for those with rare diseases.
Sarepta’s future is full of potential and possibility, but fraught with risks that could have a major impact on our operations, business plans, financial obligations, and research and development programs. We face possible regulations and approvals from the FDA and other regulatory agencies, as well as the challenge of finding, recruiting, and retaining patients for clinical trials.
Additionally, our plans may be hindered by insufficient funds or resources, manufacturing setbacks, regulatory decisions, or the ongoing COVID-19 pandemic. All of these risks are further explored in detail in our most recent SEC filings, including our Annual Report on Form 10-K. We strive to remain proactive in planning for the future and urge our investors to review all of the risks in our SEC filings that could affect the Company’s business, results of operations, and trading price of Sarepta’s common stock.