The U.S. Food and Drug Administration (FDA) recently rejected Disc Medicine’s application for bitopertin, a rare disease drug, raising concerns about the effectiveness and reliability of the Commissioner’s National Priority Voucher (CNPV) program. The rejection surprised many in the biopharmaceutical industry, as it took four months—twice as long as the estimated one to two months—and was based on information already known when the voucher was granted.
Analysts at BMO Capital Markets commented on the situation, stating in a note to investors: “The FDA’s Complete Response Letter ‘reads negatively to the broader CNPV program, showing that selection does not guarantee 1) faster reviews; or 2) approval.’” They added that although the agency initially agreed with Disc’s data package, it later changed its position. “Given issuance of the commissioners national priority voucher, the larger issue we’re seeing is inconsistency from FDA.”
Peter Pitts, president of the Center for Medicine in the Public Interest and former associate commissioner for external relations at the FDA, offered a different perspective: “Accepting a drug for a swifter review does not mean that the drug is going to be accepted,” he told BioSpace.
The CNPV program was launched in June 2025 to speed up market access for therapies aligned with U.S. national priorities. However, so far only one approval has been granted under this program—for US Antibiotics’ generic amoxicillin in December 2025—while Disc’s rejection adds uncertainty. No other decisions have been made regarding CNPV recipients, which include both smaller companies such as Achieve Life Sciences and major firms like Eli Lilly.
Truist Securities noted in a February 13 report that this decision “adds to evidence of internal agency turbulence and a meaningful shift in approval standards for rare-disease therapies.”
There are also concerns about decision-making within the FDA. Although bitopertin was reviewed by the Center for Drug Evaluation and Research (CDER), Vinay Prasad—the head of another division, Center for Biologics Evaluation and Research (CBER)—voiced opposition to Disc’s application late last year when additional review time was requested. A former FDA official questioned whether staff would feel comfortable opposing senior leadership: “There’s some question about whether or not your boss’s boss tells you, a member of the review team, to approve or not approve a drug,” they said anonymously to BioSpace.
Prasad had previously criticized accelerated approval pathways before joining CBER and appears to have played a role in this surprising rejection. The same former official remarked: “I’m not sure if this administration is even in favor of accelerated approval, in general or for rare diseases.”
Companies receiving accelerated approvals must conduct confirmatory trials afterward. Disc plans to respond with a Phase 3 trial and expects topline results by late 2026, aiming for another FDA decision by mid-2027.
Other drugs awarded priority vouchers for rare diseases include Regeneron’s DB-OTO (congenital deafness), Dompé’s Oxervate (neurotrophic keratitis), and Vertex/CRISPR Therapeutics’ Casgevy (sickle cell disease). The latter two are already approved and available on the market.
Some experts warn that being part of CNPV carries public relations risks if post-approval safety issues arise; it could appear as though drugs were insufficiently vetted.
Additionally, since CNPVs were introduced by administrative action rather than congressional legislation—unlike other FDA voucher programs—their future remains uncertain under new administrations. Patricia Zettler from Ohio State University commented: “If I were a company that received a CNPV… I would certainly ask that question.” Former CDER head Richard Pazdur echoed these concerns about consistency if political changes occur: “If we have another administration come in that may be of a different political persuasion and just change everything back, it’s just going to lead to massive inconsistencies,” he told The Wall Street Journal.
Unlike transferable rare pediatric disease priority review vouchers—which can be sold by their owners for significant sums—the non-transferable nature of CNPVs may make them less valuable for smaller biotechnology firms. Zettler noted: “FDA is calling this program a voucher program…but there’s a question what ‘voucher,’ means in these circumstances.”
Rare pediatric disease priority review vouchers remain attractive because they are transferable assets; recent sales have reached up to $150 million each.
For more information on rare pediatric disease vouchers being reauthorized through federal legislation see https://www.biospace.com/article/rare-pediatric-disease-vouchers-reauthorized-pbm-reform-funded-in-narrowly-passed-spending-bill/.
