Rocket Pharmaceuticals sold its FDA Rare Pediatric Disease Priority Review Voucher (PRV) in a $180 million sale reflecting high market demand for expedited FDA review.
Rocket received a voucher in late March for early approval of Cressradi, a hematopoietic stem cell-based gene therapy approved to treat certain children with severe leukocyte adhesion deficiency-1 (LAD-1).
The sale, which transfers PRV to an unknown buyer, extends the company’s financing runway through the second quarter of 2028, CEO Gaurav Shah, MD, said in a press release on Tuesday. Specifically, Rocket plans to use the proceeds to support advancements in its cardiovascular gene therapy pipeline.
PRVs are given with certain FDA approvals, such as drugs to treat rare diseases in children. PRV winners can use the vouchers themselves to fast-track another drug application from the standard 10-month review period to six months, but they can also be sold to other companies for immediate financial gain, which could be important for small-scale biotechs.
Rocket’s PRV sale comes months after President Donald Trump reauthorized the PRV program in conjunction with a federal funding bill that extended the program through September 2029. The reinstatement of the program was a welcome relief for rare disease drug companies, whose previous authorization had expired in December 2024, while attempts to reinstate key incentives stalled in the Senate.
In a recent legislative request to Congress, FDA Commissioner Marty McCurry, MD, proposed that Congress permanently authorize the Rare Pediatric Disease PRV Program.
“We are deeply grateful that the U.S. government continues to recognize the importance of developing treatments for rare and often devastating childhood diseases, which constitute a critical part of Rocket’s mission,” Shah said, referring to the program’s reauthorization.
There have been concerns that the PRV program could disappear forever since its creation in 2012, and the selling price of the vouchers has recently skyrocketed from their previous standard price of about $100 million. Last year, Bavarian Nordic sold the PRV associated with its chikungunya vaccine for $160 million, while Zebra Therapeutics sold the PRV of the rare lysosomal storage disorder drug Miprifa for $150 million.
Rocket’s $180 million contract signals some cooling in the PRV market, where prices recently peaked at around $200 million. So far this year, Jazz Pharmaceuticals has signed a deal to sell PRV for $200 million. Fortress Biotech’s Cyprium Therapeutics followed suit, netting a $205 windfall in its February PRV sale.
Meanwhile, FDA’s new priority voucher framework, called the FDA’s Commissioner’s National Priority Voucher (CNPV) program, is gaining momentum. The CNPV program was introduced last summer and aims to prioritize drug reviews for products that align with “U.S. national health priorities” and provides a very fast review timeline of just one to two months. Unlike traditional PRV, CNPV is not transferable.
The initiative has attracted widespread criticism and debate, but has so far led to some high-profile and quick approvals. Most recently, the CNPV program granted the first gene therapy approval for Regeneron’s Otarmeni, a treatment for inherited hearing loss that the company is offering free of charge in the United States.
