FDA announces first batch of 9 selected products for pilot projects to speed up review of “national priority” products
Gene therapy for hearing loss. A drug that helps people quit vaping or vaping. Anesthetics whose main ingredients will be produced in the United States These are among the first nine therapies selected by the FDA for a new program that will expedite regulatory review of products that meet certain national interest objectives.
Each product selected for the program receives what is known as a Commissioner's National Priority Review Voucher (CNPV). The standard drug review cycle is 10 to 12 months. The pilot program’s coupons reduce review time to one to two months. When the FDA announced the new CNPV program in June, it was unclear how products would be selected for coupons. More details now.
The FDA's Office of New Drugs consists of eight review offices designated by therapeutic area. Within each review office, therapeutic areas are further subdivided into review departments. The FDA now says each drug review unit (27 in total) is responsible for nominating products it believes meet the goals of the CNPV program. Additionally, pharmaceutical companies can apply for the program and have their requests reviewed by a designated review authority.
The FDA said the faster review will be accomplished through a team-based approach modeled after tumor boards, in which experts from different areas of oncology come together to determine the best treatment options for cancer patients. The FDA said that once all review steps for the CNPV coupon product are completed, the agency will convene a one-day tumor committee meeting to discuss the application.
The one- to two-month timeframe for regulatory decisions cannot be guaranteed. FDA reserves the right to extend a review for reasons such as incomplete applications or manufacturing violations.
“We like speed, but we don't like cutting corners on safety,” FDA Commissioner Martin Makary said in a podcast discussing nine CNPV recipients. “Throughout this entire process, our priority has been to not take any shortcuts on safety. Same people, same decisions. We reserve the right to take more time if we think more time is needed.”
Mallika Mundkur, FDA's chief medical officer and director of the CNPV program, said eligible products include those that address a substantial unmet public health need, have domestic manufacturing as a matter of national security, and equalize drug prices with other similar countries to make drugs more affordable and more accessible.
Regeneron Pharmaceuticals' DB-OTO is a gene therapy that treats a rare genetic form of hearing loss. Revolution Medicines' RMC-6236 is being developed to target pancreatic cancer. Disc Medicine's bitopertin may become the first disease-modifying therapy for the rare blood disorder erythropoietic protoporphyria. Dompé in Italy has vouchers for cenegermin (brand name Oxervate). An eye-drop version of the drug is approved to treat neurotrophic keratitis; Dompé is seeking faster FDA approval for an intranasal formulation to treat the vision loss disorder non-arteritic anterior ischemic optic neuropathy. All of these voucher recipients have the potential to address major unmet medical needs.
In other cases, domestic manufacturing is the national interest driving the issuance of coupons. Phlow received a certificate for domestic production of the active pharmaceutical ingredient (API) in the anesthetic ketamine. According to Virginia-based Phlow, there are currently no domestic suppliers of ketamine API. The old antibiotic Augmentin XR has also obtained domestic production certificates.
Other coupon recipients are EMD Serono's infertility drug Pergoveris; teplizumab (trade name Tzhield), a Sanofi drug approved to delay the onset of the most advanced types of type 1 diabetes; and Achieve Life Sciences' cytisinicline, a potential smoking cessation and smoking 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
