A gene therapy for hearing loss, an experimental pancreatic cancer treatment and seven other medications have been chosen as the first recipients of a new voucher the Food and Drug Administration is using to dramatically speed up drug reviews.
In a statement Thursday, the agency said that these nine treatments — medicines from Regeneron Pharmaceuticals, Revolution Medicines and Disc Medicine, among others — were awarded what’s been called a “national priority voucher.” These vouchers are part of a pilot program announced in June and can shorten a drug’s FDA evaluation to as little as one to two months.
Under the Prescription Drug User Fee Act, the FDA has 10 months after a company files an application to make an approval decision. The evaluation period can be compressed to six months if a company wins a “priority review,” a tool that’s been available for decades and that awards companies that bring to market treatments for rare infections or pediatric diseases a voucher that can accelerate a future drug review.
The new “national priority” program is meant to move evaluations along even more quickly, by convening a multifunctional team of physicians and scientists Commissioner Martin Makary has likened to a “tumor board” that develops a cancer patient’s treatment plan. Though selections don’t guarantee approval and reviews could take longer than two months, they should progress far more speedily, Makary said in a video posted Thursday on the social media site X.
The goal is to get decisions out fast by “reducing the idle time where applications are just sitting around not being reviewed,” he said.
Still, at the outset, the program is only open to a handful of drugs. Those products also must meet certain criteria “aligned with U.S. national priorities,” which the agency has described as addressing a U.S. health crisis, addressing unmet public health needs, making medicines more affordable, or boosting domestic drug manufacturing.
According to the FDA, each agency division was tasked to nominate a product that meets those objectives. In their video, Makary and Deputy Chief Medical Officer Mallika Mundkur walked through their reasoning.
Cytisinicline, from Achieve Life Sciences, is designed to help people quit e-cigarettes, something for which there are no FDA-approved products. DB-OTO, a gene therapy Regeneron is developing for a genetic form of deafness, has had “remarkable results” and has the potential “to change the course of a child’s life,” Mundkur said. The FDA’s oncology division found Revolution’s RMC-6236, for tough-to-treat pancreatic cancer, “potentially promising,” Makary added, and Mundkur noted how a therapy from Disc Medicine for a type of porphyria is a potentially “game-changing product.”
Makary and Mundkur cited the need to boost domestic drug production in selecting ketamine for general anesthesia and the antibiotic augmentin. They also called addressing infertility a “massive priority” of the Trump administration and a reason for choosing EMD Serono’s Pergoveris.
The awards announced Thursday are the first of two batches, the second of which could be announced in a few weeks, according to Makary. They come amidst the backdrop of a recent resurgence for biotech stocks, which have been aided by a spurt of dealmaking, positive data readouts and clarity about the administration’s drug-pricing initiatives.
The voucher program could “further reduce regulatory risk for innovative biotech,” wrote RBC Capital Markets’ Brian Abrahams, in a note to clients Friday.
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