Rocket Pharmaceuticals, a commercial-stage biotechnology company advancing genetic therapies for rare disorders, has entered into a definitive agreement to sell its Rare Pediatric Disease Priority Review Voucher for $180 million, providing non-dilutive capital to support its cardiovascular gene therapy pipeline. The transaction extends the company’s cash runway into the second quarter of 2028.
The PRV was awarded following the FDA’s accelerated approval of KRESLADI, Rocket’s gene therapy for a rare pediatric disease. A Rare Pediatric Disease PRV is granted by the FDA to sponsors of approved therapies for qualifying rare pediatric conditions and may be used to obtain priority review for a subsequent marketing application or sold to another sponsor. The PRV program was reauthorized in February 2026, extending the availability of vouchers for qualifying therapies.
Proceeds from the transaction will support the advancement of Rocket’s cardiovascular gene therapy pipeline, which includes three clinical-stage programs targeting the major inherited cardiomyopathy subtypes. These programs address Danon disease, PKP2-associated arrhythmogenic cardiomyopathy, and BAG3-associated dilated cardiomyopathy, conditions that together represent more than 100,000 patients in the United States and European Union.
Rocket Pharmaceuticals trades on Nasdaq under the ticker RCKT and supports its pipeline with proprietary AAV manufacturing capabilities and multi-year efficacy and safety data in cardiac gene therapy.
KEY QUOTE:
“The monetization of our PRV, following the FDA approval of KRESLADI, provides meaningful non-dilutive capital and extends our cash runway into the second quarter of 2028. This strengthens our ability to advance key clinical milestones across our cardiovascular gene therapy pipeline, with all programs on track. We are deeply appreciative of the U.S. government’s continued recognition of the importance of therapeutic development for rare and often devastating pediatric disorders, which comprises an essential part of Rocket’s mission.”
Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharmaceuticals
