Bitopertin

iStock, Khafizh Amrullah The FDA’s Commissioner’s National Priority Voucher program, unveiled in June 2025, is “shrouded in secrecy,” Democratic representative Jake Auchincloss said last month, as regulatory and biopharma leaders try to decode the criteria for investigational or approved drugs to receive a voucher. Shortening drug review times by a factor of 10, a Commissioner’s National Priority Voucher would seem to be highly valuable to biopharma companies looking to bring investigational assets to market. But experts say that the selection process for the program remains murky, raising challenges for potential applicants and adding to concerns about the FDA’s overall transparency. The FDA has failed to respond to several inquiries from Congress about the CNPV program, Rep. Jake Auchincloss (D-MA) said, according to reporting from the Associated Press in early February. The congressman wrote directly to the agency stating his concerns about how the program has been “shrouded in secrecy.” “The public must have transparency about the ‘voucher’ program, under which drug approvals have been made almost wholly and in an unprecedented manner by the FDA’s political leadership,” he wrote. The program has had highlights, however. Johnson & Johnson’s Tecvayli/Darzalex combo, which received a voucher in December 2025, won FDA approval earlier this month to treat patients with multiple myeloma after cutting the risk of death from all causes by 54% in a Phase 3 trial. By all accounts, this was a victory—and a national priority. “In theory, the benefits look great,” Kinnari Patel, CEO of think tank RTW Institute, told BioSpace of the scheme, which was introduced in June 2025. “The idea of any drug that has a huge unmet medical need or need in the American population to be reviewed in one or two months, is great, full stop.” But the reasons for the FDA’s selection of other CNPV recipients remain obscure. For example, Patel noted, while FDA Commissioner Marty Makary has said that product selection is based on unmet medical need, “we don’t know what that means.” This makes it hard for companies to determine if they should apply for a voucher. “The criteria for selection is not as transparent and standardized as we would like,” Patel concluded. Specifically, “what’s unknown is [that] the decision is really made by the commissioner and the commissioner’s office.” Again, regulatory experts have sounded the alarm over what multiple sources have called ‘fiat’ decision-making at the agency more generally, most recently in relation to the FDA’s initial refusal to review Moderna’s mRNA-based flu vaccine. The agency ultimately reversed course, but not before President Donald Trump reportedly had a word with Makary . FDA FDA’s Circle of Trust Is Shrinking, Making Many Decisions Feel Like ‘Fiat’ FDA decisions lack majority consensus, experts agree, possibly leading to less nuanced verdicts on new drug applications. This type of “fiat” decision-making, as multiple regulatory experts have called it, is also bleeding into the agency’s policymaking. March 3, 2026 · 4 min read · Heather McKenzie Read more Guidance Required—Again In announcing the CNPV program last summer, the FDA described a reduction in its review times from 10–12 months to 1–2 months for investigational products that align with certain national priorities. These include addressing a U.S. public health crisis, increasing affordability and onshoring drug development and manufacturing—a major push during the first year of President Donald Trump’s second term. So far, the FDA has approved four products under the program, all within the promised two-month window: Novo Nordisk’s Wegovy HD , a 7.2 mg injectable dose of semaglutide, J&J’s Tecvayli/Darzalex combo, USAntibiotics’ Augmentin XR —an extended release formulation of a combination of amoxicillin and the β-lactamase inhibitor clavulanate potassium first approved in 2002 —and Boehringer Ingelheim’s Hernexeos for non-small cell lung cancer. The agency has also rejected one candidate, Disc Medicine’s rare disease asset bitopertin. This decision took four months to materialize. This leaves at least 13 outstanding vouchers, with decisions for Sanofi’s Type 1 diabetes drug Tzield and Eli Lilly’s obesity pill orforglipron still pending. The more commonly cited concern is not the missed deadlines but a lack of transparency surrounding the CNPV program. This echoes a recurring trend this past year. Biopharma and political leaders have questioned the clarity of many other FDA programs, including the recently announced one-pivotal trial policy and Plausible Mechanism Pathway, as well as decisions around products, including uniQure’s gene therapy for Huntington’s disease and Sarepta’s Duchenne muscular dystrophy gene therapy Elevidys . Huntington’s disease FDA Official Fires Back at UniQure, Doubles Down on Sham-Controlled Trial UniQure does not have to drill placebo burr holes in the skulls of patients with Huntington’s disease, an unnamed FDA senior official said on Thursday. Instead, the company would anesthetize them and put “one to three nicks” in their scalp. March 6, 2026 · 2 min read · Tristan Manalac Read more There have not been any guidance documents, regulations or statutory requirements revealed to broadly define the CNPV program, “which normally happens with any regulations with FDA,” Patel noted. “From a process perspective, there’s no FDA regulations” that codify CNPVs, just a press release posted to the agency website. The FDA put out a call for applications on its website and began accepting them last July. A few months later, it announced the first round of CNPV awardees. The inaugural group included Sanofi’s Tzield for type 1 diabetes, Revolution Medicines’ RMC-6236 for pancreatic cancer and Disc Medicine’s bitopertin for erythropoietic protoporphyria—an ultrarare skin disease that causes extreme sensitivity to the sun. The second batch of CNPVs, revealed the following month, caused some head scratching, as the only investigational drug on the list was Eli Lilly’s oral weight loss candidate orforglipron. The rest were already on the market. In fact, of the vouchers that have been granted to date, fewer than one-third fit the description as Jama Pitman understood it. “About five or so . . . were true investigational new agents that didn’t have an approval in the U.S. or outside the U.S., and they were for things that you would think there’s an urgent unmet medical need,” Pitman, a regulatory strategy lead at psychedelic research organization Rose Hill Life Sciences, told BioSpace . The products accepted into the program so far represent “a broad component” of conditions, Patel said, including infertility, type 1 diabetes, deafness and obesity. Augmentin XR “met sort of the onshoring and national security [criterion], so just making sure that we’re able to manufacture a needed antibiotic,” Pitman said. But some selections have raised eyebrows. Patel was “surprised” to see the November selection of Hernexeos, which was first approved by the FDA in August 2025 for patients with HER2-mutant advanced non-small cell lung cancer (NSCLC). As there are already several treatments on the market for NSCLC, she would have expected a product for small cell lung cancer (SCLC), in which there is higher unmet need, to have taken precedence. Hernexeos won FDA approval last August for patients who had received prior systemic therapy. The new approval opens up first-line treatment. As for Disc’s bitopertin, Pitman said she was “a little confused” as to why this product was included in the pilot program, as Disc had already filed a new drug application for the drug for erythropoietic protoporphyria prior to receiving the CNPV. She also pointed out that Disc was seeking accelerated approval, which relies on a surrogate endpoint, though J&J’s approval was also of the accelerated variety. “Given the need to carefully evaluate the surrogate endpoints as reasonably likely to confer clinical benefit . . . an [approximate] two-month review of the data would seem rushed, and the agency likely would be unable to properly interrogate the data to make an informed assessment of the benefit/risk profile,” Pitman said. Indeed, it took the FDA four months to return a verdict to Disc. And when it did, it was not the answer the company had hoped for. Analysts at Truist Securities called the rejection “surprising” in a Feb. 13 note, “given that bitopertin met “in our view, all key criteria for accelerated approval.” The decision also “reads negatively to the broader CNPV program, showing that selection does not guarantee 1) faster reviews; or 2) approval,” BMO Capital Markets wrote in a February 13 note. But Peter Pitts, president of the Center for Medicine in the Public Interest, said the rejection is a sign that this aspect of the program is working. “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 last month. “[FDA is] choosing ones that are interesting, that are different. There is no shoo-in.” Compass Left Out Not every company that applied for a CNPV got one. In early February, news broke that Compass Pathways’ psilocybin therapy COMP360 had been on the short list for a voucher but was ultimately not chosen. The FDA had included COMP360 as part of the initial October list, Pitman said, meaning HHS decided not to award the psychedelic treatment a CNPV. “It’s not overly surprising that that happened,” Pitman conceded. The majority of the CNPV recipients had a safety database that was relatively known and efficacy that had already been established, while no psychedelic has ever won FDA approval. Additionally, psychedelics are generally going to come with extensive Risk Evaluation and Mitigation Strategy (REMS) programs upon the first approval, not to mention needing to be reclassified by the DEA, as well as individual states, Pitman noted. “I gave a lot of kudos to the FDA and the commissioner for putting [COMP360] on the list,” Pitman said. But, she added, “two months, quite frankly, is probably not enough time.” .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' }); } });