The Senate failed to pass a massive spending bill on Thursday—which includes the rare pediatric PRV program but also funding for the Immigration and Customs Enforcement’s large-scale crackdown in Minnesota and other states.
The delay of reauthorizing the rare disease priority review voucher program has become an unexpected headache for biotech leaders, who have been lobbying the government to get the program moving again.
“We think it’s absolutely critical to support innovative research, to support pediatric, rare disease medicines coming forward,” Ionis CEO Brett Monia told BioSpace at the J.P. Morgan Healthcare Conference earlier this month.
The Senate failed on Thursday to pass a massive spending bill that included the rare pediatric PRV program. It also included funding for the Department of Homeland Security (DHS), under which the Immigration and Customs Enforcement (ICE) is executing its large-scale crackdown in Minnesota and other states. The vote was 55–45 against the bill.
While President Donald Trump and Senate Democrats reached a new deal on Thursday that separates the DHS funding from that of other departments, House Speaker Mike Johnson said his chamber is unlikely to return to vote on the deal before Monday, Bloomberg reported Friday, almost assuring a partial government shutdown over the weekend.
Meanwhile, biopharma companies with PRV-eligible programs are preparing for either eventuality.
Monia said Ionis was already racing to get zilganersen submitted for FDA review as fast as possible to help patients with Alexander disease who have few options. But the prospect of losing out on a potential priority review voucher that could support the company’s next rare disease treatment added another flame to the fire.
Under a temporary extension, rare disease drugs approved by Sept. 30, 2026, will still qualify for a PRV.
“Getting the drug to patients, of course, is number one. But not losing out on a potential PRV is one of our other objectives,” Monia explained. “Our team has worked through the holidays to get the NDA submitted as quickly as possible to meet that deadline.”
PRVs can be sold, with most recent figures valuing them at around $150 million. Earlier this month, Jazz Pharmaceuticals revealed it had sold a voucher for $200 million to an unnamed buyer. Sometimes companies keep them—Ionis has numerous programs in development that a PRV could be applied to. But many smaller companies often choose to sell them and reinvest the money.
The fate of the PRV program will also have an effect on mid-sized companies with broader R&D pipelines.
“The PRV directly impacts how many of our rare-disease shots on goal we can fund and move into the clinic,” Justin To, CEO of skeletal dysplasia programs at BridgeBio, told BioSpace earlier this month.
Catherine Owen Adams, CEO of Acadia Pharmaceuticals, has been lobbying the government on the PRV issue as well. She credits the program with accelerating investment in the rare disease space.
“Since that’s come into being, we’ve seen an acceleration of rare drug approvals because the innovation has been worthwhile in terms of the large investment for small patient populations,” Owen Adams said.
Monia was clear to state that this is an issue that Congress has to solve, not the FDA. Both he and Owen Adams said that FDA leadership has signaled nothing but support for the program. Monia met with FDA Commissioner Marty Makary in San Diego last year when the chief regulator was touring the country. Monia said PRVs came up in the meeting, and that Makary indicated that the program has his support.
Owen Adams said the holdup has mainly been about finding the right legislation to pin the PRV reauthorization to.
“There does seem to be widespread understanding and support that this is a good thing for rare disease patients, as well as American innovation,” she said, speaking to her experience lobbying on Capitol Hill to Republicans and Democrats alike. “I feel vaguely positive that it is going to be renewed. But, you know, stranger things have happened.”
With a government shutdown looming over the omnibus bill, rare disease biotechs may have to wait even longer for certainty.
