Cytisinicline

FDA Commissioner Marty Makary unveiled the first nine companies to win his new National Priority Vouchers, a bigger cohort than originally planned as part of the agency’s effort to significantly cut some drug review times. The voucher program aims to review selected drugs in as little as one month after submission, instead of the typical 10 to 12-month timeline. When it was first announced in June, the FDA said up to five companies would participate in the first year, according to a webpage screenshot from July. That language has since been removed from the FDA’s site . Makary has vowed to release even more vouchers in the coming weeks, aligning with President Donald Trump’s priorities for lowering drug prices and increasing US manufacturing. “We are committed to accelerating the approval of products that advance critical national health priorities and lowering the costs of drugs for the American people,” HHS spokesperson Andrew Nixon said in a statement on Friday when asked about the wider scope of the program. “That commitment remains unwavering.” The voucher announcement was folded into a broader event at the White House on Thursday where Trump announced sweeping new initiatives to increase access to IVF treatment, including a new “most favored nation” pricing deal with EMD Serono. That company’s next-gen fertility treatment Pergoveris is one of the nine products selected. Other recipients of the vouchers include Regeneron and Sanofi. Makary told Endpoints News after the event that more recipients will be announced “on a rolling basis.” He said in an associated FDA Direct video that the next batch will be unveiled in a couple of weeks. Makary said at the event that the voucher selections were based on a trio of factors, namely unmet need, domestic manufacturing efforts and price. They also address a range of Makary’s priorities, from jumpstarting certain key API production in the US to finding new treatments for notoriously difficult-to-treat conditions like pancreatic cancer. One of the selections for a voucher is Regeneron’s new gene therapy for an inherited type of deafness in children, which had positive results from a small trial published Sunday in the New England Journal of Medicine . While Makary said that the price of Regeneron’s gene therapy would be low, a Regeneron spokesperson told Endpoints it’s still “too early for a pricing decision.” The drugs also range across the development timeline, with ones from Revolution and Achieve Life Sciences still waiting on an initial approval. Mark Rubinstein, interim chief medical officer at Achieve, said the selection of cytisinicline for vaping cessation shows the FDA’s “strong commitment to addressing the urgent public health challenge posed by nicotine dependence.” Others given a voucher, including Sanofi’s autoimmune type 1 diabetes drug teplizumab, are already approved in other indications, so the voucher will likely speed up their supplemental application processes. That’s also the case for Dompé, which received a voucher for its intranasal eye medication cenegermin-bkbj to treat a condition disrupting blood flow to the optic nerve. The drug is already approved for neurotrophic keratitis. Dompé Chief Development Officer Ahmed Enayetallah said the FDA’s ophthalmology division first approached the company about including the product in the voucher pilot. Still, the company’s focus is on executing a Phase 3 pivotal program that will launch soon. “At the end of the day, it’s the totality of the package that will be looked at,” Enayetallah said. “I don’t think the decision will be made on the partial dataset.” Disc Medicine got a voucher for its investigational drug bitopertin to treat erythropoietic porphyrias, a rare disease that limits people’s ability to produce red blood cells, which can be painful and force them to avoid sunlight. CEO John Quisel told Endpoints that bitopertin will be made in the US, which he believes “was one of the pillars that we were judged on.” Quisel said the company submitted its voucher application in August, ahead of a late September new drug application. It received word from the FDA on Thursday a few hours before the announcement. The voucher could add pressure on the company, which now may need to be ready to launch the product before the end of the year. The company had not yet received a PDUFA date after submitting its NDA and before receiving the voucher. “We’re going to have a one to two week planning process, but the base expectation is we’re accelerating everything … from manufacturing to the commercial team,” Quisel said.

Revolution Medicines and Disc Medicine are among the recipients of a Commissioner’s National Priority Voucher. \n 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. 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.