The FDA has handed out the first nine national priority vouchers, positioning companies including Merck KGaA, Regeneron and Sanofi to complete the approval process in one to two months.
FDA Commissioner Marty Makary, M.D., launched the program in June, and the agency began accepting applications in July. The program is designed to cut product approval times, which are typically 10 to 12 months, for medicines that align with U.S. national priorities. Specific priorities include addressing the U.S. public health crisis, treating large unmet medical needs and delivering transformative treatments.
The first nine recipients of a Commissioner’s National Priority Voucher (CNPV) are a mix of approved drugs and investigational medicines. The investigational products include DB-OTO, the gene therapy that Regeneron is developing to treat a rare genetic form of hearing loss.
Regeneron plans to seek FDA approval this year. If the CNPV works as intended, the timeline suggests the gene therapy could come to market early next year at the latest. The company has published data on the gene therapy in 12 patients, nine of whom only received the treatment in one ear.
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Talking on an FDA podcast, Mallika Mundkur, M.D., who leads the CNPV program at the agency, said the DB-OTO data are “pretty remarkable” but cautioned that “we need to learn more about” its safety. The podcast was hosted by Makary, who said the hearing restoration data point to the need for the FDA “to get a decision out promptly using the standard scientific process, cutting any wasted time.”
Makary added that “we’re hearing the company may make it available either at an extremely low price or for free, which of course is part of a national priority to make drugs more affordable.” The criteria for the CNPV program include lowering the U.S. price of a drug consistent with most-favored nation pricing.
Revolution Medicines’ RMC-6236 and Disc Medicine’s bitopertin are among the other recipients of a CNPV. RMC-6236, also called daraxonrasib, is a RAS-MULTI(ON) inhibitor that is in phase 3 development as a treatment for forms of pancreatic and non-small cell lung cancer. Data from one of the trials are due next year. Revolution is holding off on adjusting its timelines while it assesses the impact of the voucher.
Discussing RMC-6236 on the podcast, Makary said the FDA’s oncology division “thought this is something that is potentially promising.” The FDA commissioner went on to say the agency is going into its divisions and asking reviewers what programs they think “may potentially look amazing.” Makary framed the talks as a push for the FDA to move beyond “receive-only mode” when identifying assets it can accelerate.
Meanwhile, Disc’s bitopertin is a GlyT1 inhibitor being assessed in rare, genetically driven hematologic diseases. The biotech filed for accelerated FDA approval of the small molecule last month. Disc originally applied for a priority review that would reduce the review time from 10 months to six months.
The FDA also awarded CNPVs to Merck’s pergoveris and Sanofi’s teplizumab. Pergoveris, a fertility drug, is already available in 74 countries. Merck plans to file for FDA approval of the drug using its CNPV, which it disclosed as part of a broader deal with the U.S. government to expand access to fertility treatment. Teplizumab, which Sanofi sells as Tzield, is FDA-approved to delay the onset of forms of Type 1 diabetes.
Officials gave another two CNPVs to cytisinicline for nicotine vaping addiction and cenegermin-bkbj for blindness. Achieve Life Sciences is seeking approval of cytisinicline for smoking cessation. Cenegermin is already sold as Oxervate by Dompé Farmaceutici to treat a corneal disease.
The final two CNPVs are focused on onshoring, with the FDA using the vouchers to try to boost domestic production of ketamine for general anesthesia and of the antibiotic augmentin XR. Phlow received the ketamine CNPV.
