Orforglipron

iStock, beast01 Priority voucher awardees and regulators could feel pressure to “meet the moment” as FDA watchers question the intent and feasibility of the Commissioner’s National Priority Voucher program. For the first 15 awardees of the FDA’s new Commissioner’s National Priority Voucher program, the pressure to succeed is palpable. “It’s super exciting,” John Quisel, CEO of Disc Medicine, told BioSpace . “It is kind of a jolt as well—a feeling of responsibility, of okay, we’re a national priority now to get this drug to these patients. We can’t fail, right?” Launched in June, the Commissioner’s National Priority Voucher (CNPV) program is intended to shorten the timeline for drug review from 10–12 months to one-to-two months. The vouchers are granted to companies that are “aligned with U.S. national priorities,” according to the announcement, including those addressing a health crisis, delivering more innovative cures, addressing an unmet public health need or increasing domestic drug manufacturing as a national security issue. The first nine vouchers were issued on October 16, followed by six more on November 6. So far, indications have included type 1 diabetes, infertility, congenital deafness, obesity and sickle cell disease. The receiving companies are as big as Johnson & Johnson and as small as Disc, which sports a market cap of a modest $3.24 billion, compared to J&J’s more than $470 billion. When the FDA put out a call for proposals for CNPVs this summer, “We felt like we have a rare disease, high unmet medical need, a potential therapy that’s addressing an underlying cause of disease . . . so we put in an application,” Quisel said, describing the company’s drug candidate for erythropoietic porphyrias (EPP). He added that Disc also had “support and positivity” in interactions with the dermatology review division at the agency. “I think that was an important part of feeling like there was a rationale for a successful bid for one of these national priority vouchers.” The first nine awardees—five of which stemmed from company applications, while four were nominated by agency reviewers, according to STAT News —were all investigational therapies. Some, such as Disc’s bitopertin , now have regulatory filings awaiting FDA review, while others are still in clinical trials. “One thing that struck me is how disparate they are, particularly across stage of development,” Eva Temkin, partner at the law firm Arnold & Porter, said of the first nine awardees. “You have some that are very close to the end, and you have some that are still in Phase III.” Then, five of the six vouchers announced in round two went to therapies already on the market. Notably, these were announced immediately following a White House press conference to unveil a drug pricing agreement for GLP-1 medicines, including Novo Nordisk’s blockbuster weight loss drug Wegovy as well as Eli Lilly’s investigational obesity pill orforglipron, each of which received a CNPV. “This was not, as I understand it, a prioritization exclusively based on public health imperative, which historically has been the purview of FDA in the way that these have been made,” Temkin, who previously served as acting policy staff director at the FDA’s Office of Therapeutic Biologics and Biosimilars, told BioSpace . “You have criteria that have not been well defined but arguably have expanded beyond that purview.” Regulatory Wegovy, Casgevy Among Latest FDA Priority Review Voucher Recipients The FDA awards a second round of Commissioner’s National Priority vouchers to six larger biopharma companies. And this time, with the exception of Eli Lilly’s orforglipron, the vouchers are for drugs that are already on the market. November 6, 2025 · 3 min read · Dan Samorodnitsky Courting Controversy Modeled after FDA Commissioner Marty Makary’s experience as a surgical oncologist, the review process for CNPV recipients will involve experts from across the agency gathering to review the chosen candidates in a one-day “tumor board style” meeting, according to the FDA’s June press announcement . The priority vouchers have reportedly been a source of conflict among Center for Biologics Evaluation and Research Chief Vinay Prasad and his former counterpart at the Center for Drug Evaluation and Research, George Tidmarsh, who resigned earlier this month amid a probe into his “personal conduct” at the agency. Tidmarsh raised questions about the legality of the program, particularly the one-day meeting format, according to reporting by STAT News . The FDA in its press release announcing the second round of vouchers appeared to address this issue, writing: “Under the Federal Food, Drug, and Cosmetic Act; the 21st Century Cures Act; and the Food and Drug Administration Safety and Innovation Act, the FDA is authorized to test innovative regulatory approaches to accelerate review, foster public health preparedness, and improve access to safe, effective, and affordable therapies.” Steven Grossman, policy and regulatory consultant and author of the FDA Matters blog, has also questioned the program. “I was initially very unhappy about it,” Grossman said during a recent BioSpace webinar , “because the whole idea is that priority at FDA should be built around unmet medical need, and by traditional standards that meant serious and life-threatening diseases, and it was clear that’s not what they were focused on.” He came around, however, when he broadened his definition of unmet medical need to include things like shortages. “I’ve looked at, for instance, what was the rationale for all nine of the choices and in all nine cases I could make a case that was unmet medical need,” he said of the initial awardees. Responding to BioSpace ’s request for comment on the second tranche of awardees, Grossman said in an email: “So far, we have less information about the new set and their intended use. Hopefully, they will meet the criteria I applied to the first batch: they address unmet medical needs that are legitimately on FDA’s priority list and can be justified without reference to separate dialogues about pricing and other national (but not necessarily FDA) priorities.”   A High Unmet Need For Disc and the EEP community, the unmet need is about as traditional as it gets. Bitopertin is a glycine transporter 1 inhibitor in confirmatory studies for EPP—a family of rare, debilitating and potentially life-threatening diseases caused by mutations that affect the heme synthesis pathway. In EEP, the partial defect occurs in the last enzyme, leading to the buildup of a chemical called protoporphyrin IX (PP9), which absorbs sunlight and generates dangerous free radicals, Quisel explained. Ultimately, patients spend their lives avoiding the sun. This build-up of PP9 can also lead to gallstones, gallbladder removal and liver damage. .responsive-container { display: flex; flex-wrap: wrap; border: 1px solid #ededed; font-family: Helvetica, Arial, Sans-Serif; padding: 20px 20px 10px 20px; } .column-left { flex: 1; max-width: 45%; padding: 20px 20px 10px 20px; } .column-right { flex: 2; padding: 20px 20px 10px 20px; } @media (max-width: 768px) { .column-left, .column-right { max-width: 100%; flex: 100%; padding: 10px 0; } } Subscribe to ClinicaSpace! Clinical trial results, research news, the latest in cancer, cell and gene therapy hbspt.forms.create({ region: "na1", portalId: "4413123", formId: "674f4dc0-7371-4190-86cc-e1afd00b08ea", onFormSubmitted: function($form, data) { window.dataLayer = window.dataLayer || []; window.dataLayer.push({ 'event': 'GTMevent', 'event_name': 'newsletter_sign_up' }); } }); Quisel noted that Disc was “right on the cusp of submitting our [new drug application] anyway, so we knew that we would be providing the FDA with what we think is a complete file.” Disc filed an NDA seeking accelerated approval of bitopertin in EEP in September. Assuming accelerated review, Disc had anticipated launching bitopertin “sometime in the second half” of 2026, Quisel said. Now with a commissioner’s voucher in hand, the biotech is looking at potentially having a drug on the market late this year or early in 2026. “We were already building a lot of the executive functions around commercialization, selling a drug,” the CEO said. “But everything now has slipped into high gear to meet the moment.” However, according to Temkin, Disc—and the EEP community’s gain—could be a sticking point for other companies and patients, as the priority vouchers could potentially slow the path to market for other investigational drugs. “It is extremely resource intensive to do the kind of expedited review that is being imagined here, to the extent that we understand the contours of it,” she said. “I think the biggest con is probably that all of that attention by review teams means they’re not engaging on other applications. The pie doesn’t just get bigger.” The CNPV awardees are likely not the only ones who could feel they must deliver under this new program; so too could FDA reviewers. “There is a huge amount of pressure to approve or take action on something quickly, that can result in a less creative approach,” Temkin said. She suggested there could be less “back and forth” on the drug’s final label, and less opportunity to correct small problems along the way. During a typical six-month priority review period, there are usually a “series of meetings,” Temkin explained, during which the FDA has an opportunity to resolve questions. “If you have an extremely condensed review period, I suspect there’s less opportunity for that kind of engagement.”