FDA vouchers are normally a coveted prize for biopharma companies, but a surprise rejection for Disc Medicine’s rare disease drug has biopharma reconsidering.
The FDA’s rejection of Disc Medicine’s rare disease drug bitopertin came as a surprise to the entire biopharma world—including, perhaps, other holders of a coveted Commissioner’s National Priority Voucher. Not only did the rejection take four months to materialize—twice as long as the 1–2 month estimate—but it was based on information known when the voucher was awarded.
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,” analysts at BMO Capital Markets wrote in a note to investors Friday morning following the rejection. The firm further noted that the agency had initially agreed to Disc’s submitted data package and then apparently changed its mind. “Given issuance of the commissioners national priority voucher, the larger issue we’re seeing is inconsistency from FDA.”
For Peter Pitts, president of the Center for Medicine in the Public Interest, however, the rejection is a sign that “the system works.
“Accepting a drug for a swifter review does not mean that the drug is going to be accepted,” Pitts, a former associate commissioner for external relations at the FDA, told BioSpace in an interview.
The Commissioner’s National Priority Voucher (CNPV) program, launched in June 2025, was designed to accelerate to market therapies that are aligned with U.S. “national priorities.” But so far, the program has not quite lived up to expectations. One approval has gone through—US Antibiotics’ application for a generic amoxicillin in December 2025—and now Disc has been hit with a rejection. Otherwise, no other decisions have been made on any CNPV awardees. That includes smaller companies like Achieve Life Sciences and giants like Eli Lilly.
The Disc decision “adds to evidence of internal agency turbulence and a meaningful shift in approval standards for rare-disease therapies,” Truist Securities wrote in a February 13 note.
The process and rejection of Disc’s drug also highlights concerns about unilateral decision making. Bitopertin was reviewed through the Center for Drug Evaluation and Research (CDER), yet it was Center for Biologics Evaluation and Research (CBER) head Vinay Prasad, who signaled opposition to Disc’s application in December 2025, when a review team asked for more time to complete its work.
“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,” a former FDA official told BioSpace on the condition of anonymity. “Would you do that? It can take a lot for someone to say no.”
Prasad’s opinion could be just a one-off on Disc’s application, but it could signal trouble for other drugs as well. In his time before taking the CBER chief position, Prasad had been highly critical of the FDA’s use of accelerated approval pathways, particularly regarding surrogate endpoints, and now it seems he has led a surprising rejection of a drug that was under review using such a pathway.
“I’m not sure if this administration is even in favor of accelerated approval, in general or for rare diseases,” the former official said.
Companies who receive an accelerated approval have to follow up with a confirmatory trial. Disc is planning to address the FDA’s rejection with a Phase 3 trial, with topline data expected by the end of 2026. That would put Disc in line for another decision by mid-2027.
As for rare diseases, other drugs so far awarded priority vouchers in this space include Regeneron’s DB-OTO for congenital deafness, Dompé’s Oxervate for neurotrophic keratitis and Vertex and CRISPR Therapeutics’ Casgevy for sickle cell disease. The latter two products are already on the market.
Questions That Need Answering
With all of this in mind, should a biopharma company be excited to be a part of the CNPV program? It’s not clear—for starters, there’s a potential public relations risk inherent in winning a voucher, the anonymous former FDA official suggested. If a drug approved through the program were to cause an adverse event in a patient once it was on the market, it could seem as if the drug “didn’t get vetted properly,” they said.
In addition, because the program was simply announced by the FDA and not enacted in congressional legislation, like other FDA voucher programs, it is “an open question” whether the agency under the next administration will even honor the vouchers, Patricia Zettler, professor at the Moritz College of Law at Ohio State University, told BioSpace in an interview. “If I were a company that received a CNPV,” she added, “I would certainly ask that question.”
Even former CDER head Richard Pazdur has questioned the quality and longevity of the CNPVs. “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 said in an interview with The Wall Street Journal last week.
Because CNPVs are not transferable, existing vouchers like the rare pediatric disease priority review vouchers (PRVs) may be more prized for smaller biotechs. PRVs can be sold by their owners, with recent prices reaching as much as $150 million. Not enacted in statute, CNPVs may be closer to a simple promise by the FDA to review drugs faster.
“FDA is calling this program a voucher program,” Zettler said, “but there’s a question what ‘voucher,’ means in these circumstances.”
