A gene therapy for hearing loss. A drug that helps people quit e-cigarettes or vaping. An anesthetic whose main ingredient will be manufactured in the U.S. These are among the first nine therapies selected by the FDA for a new program that will accelerate regulatory review of products that meet certain national interest goals.
Each product selected for this program was awarded what’s called a Commissioner’s National Priority Review Voucher (CNPV). Standard drug review is 10 to 12 months. The vouchers from this pilot program shorten reviews to one-to-two months. When the FDA announced the new CNPV program in June, it was unclear how products would be selected for a voucher. There’s more detail now.
The FDA’s Office of New Drugs is comprised of eight review offices designated by therapeutic area. Within each review office, the therapeutic area is broken down further into review divisions. The FDA now says each drug review division — there are 27 total — has been charged with nominating a product it believes meets the goals of the CNPV program. In addition, drug companies may apply to the program and have their request reviewed by the designated review division.
Speedier review will be accomplished with what the FDA describes as a team-based approach emulating a tumor board, a practice in which specialists from different areas of oncology convene to determine the best course of treatment for a cancer patient. The FDA said that once all of the review steps for a CNPV-voucher product are complete, the agency will convene a one-day tumor board style meeting to discuss the application.
The one-to-two month timeframe for a regulatory decision is not guaranteed. The FDA reserves the right to extend the review for reasons such as an incomplete application incomplete or manufacturing violations.
“We like speed, but we don’t like cutting any corners on safety,” FDA Commissioner Martin Makary said in a podcast discussing the nine CNPV recipients. “That is our number one priority throughout this is there are no corners cut on safety. It’s the same people, same decisions. We reserve the right to take more time if we believe it needs more time.”
Mallika Mundkur, chief medical officer of the FDA and the official in charge of the CNPV program, said eligible products include those addressing a large unmet public health need, domestic manufacturing as a national security issue, and equalizing drug prices with other comparable nations to make medicines more affordable and accessible.
Regeneron Pharmaceuticals’ DB-OTO is a gene therapy that could address a rare, genetic form of hearing loss. Revolution Medicines’ RMC-6236 was developed for pancreatic cancer. Disc Medicine’s bitopertin could become the first disease-modifying treatment for the rare blood disorder erythropoietic protoporphyria. Italy-based Dompé has a voucher for cenegermin (brand name Oxervate). The eye drop version of this drug is approved for treating neurotrophic keratitis; Dompé is seeking speedier FDA approval of an intranasally administered formulation to treat the vision-loss disorder non-arteritic anterior ischemic optic neuropathy. All of these voucher recipients offer the potential to address major unmet medical needs.
In other cases, domestic manufacturing is the national interest driving the award of a voucher. Phlow received a voucher for domestic production of the active pharmaceutical ingredient (API) in the anesthetic ketamine. According to Virginia-based Phlow, there are currently no domestic ketamine API suppliers. The old antibiotic Augmentin XR from USAntibiotics also received a voucher for domestic manufacturing.
The other voucher recipients are infertility drug pergoveris from EMD Serono; teplizumab (brand name Tzhield), a Sanofi drug approved for delaying the onset of the most advanced forms of type 1 diabetes; and Achieve Life Sciences’ cytisinicline, a potential smoking- and vaping-cessation drug.
The FDA said it expects to announce another group of voucher recipients in the coming months.
Photo: Tom Williams/CQ-Roll Call, via Getty Images
