Rocket Pharmaceuticals has offloaded its lucrative FDA rare pediatric disease priority review voucher (PRV), striking a $180 million sale that reflects the high market demand for a speedy FDA review.
Rocket earned its voucher from its late March accelerated approval of hematopoietic stem cell-based gene therapy Kresladi, which was cleared to treat certain children with severe leukocyte adhesion deficiency-1 (LAD-1).
With the sale, which transferred the PRV to an unknown buyer, the company extends its cash runway into the second quarter of 2028, CEO Gaurav Shah, M.D., noted in a Tuesday press release. Rocket specifically plans to use the proceeds to support the advancement of its cardiovascular gene therapy pipeline.
PRVs are given alongside certain FDA approvals, including for rare pediatric disease drugs. While PRV awardees can use the voucher themselves to fast-track another drug application from the standard 10-month review period to six months, they are sometimes sold to other companies, given the immediate monetary return, which can be crucial for smaller biotechs.
Rocket’s PRV sale comes a few months after President Donald Trump reauthorized the PRV program alongside a government funding bill, extending the program through September 2029. The revival of the program was a welcome relief for rare disease drugmakers, as its previous authorization had let up in December 2024 while attempts to bring back the key incentive had stalled in the Senate.
In his recent legislative request to Congress, FDA Commissioner Marty Makary, M.D., proposed that Congress permanently authorize the rare pediatric disease PRV program.
“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,” Shah said, referring to the program reauthorization.
Amid concerns that the PRV program would be gone for good following its 2012 establishment, sale prices of the vouchers have recently spiked from the previous standard value of around $100 million. Last year, Bavarian Nordic sold a PRV tied to its chikungunya vaccine for $160 million, while Zevra Therapeutics handed off its PRV from rare lysosomal storage disorder drug Miplyffa for $150 million.
Rocket’s $180 million deal marks a slight cooling in the PRV market, which recently saw prices peak around $200 million. So far this year, Jazz Pharmaceuticals inked a deal to sell its PRV for $200 million. Fortress Biotech’s Cyprium Therapeutics followed suit, earning a $205 windfall from its PRV sale in February.
Meanwhile, the FDA’s new priority voucher framework, called the FDA's Commissioner's National Priority Voucher (CNPV) program, is picking up steam. The CNPV program was introduced last summer and looks to prioritize drug reviews for products that align with “U.S. national health priorities,” offering an extra speedy review timeline of just one to two months. Unlike traditional PRVs, a CNPV cannot be transferred.
Although the initiative has attracted a swath of criticism and discourse, it has so far resulted in several high-profile speedy approvals. Most recently, the CNPV program awarded its first approval for a gene therapy in Regeneron’s genetic hearing loss therapy Otarmeni, which the company is offering for free in the U.S.