FDA Accelerates Review Process for Nine Innovative Therapies

The U.S. Food and Drug Administration (FDA) has selected nine innovative therapies for its new pilot program aimed at expediting the regulatory review process for products deemed to meet national interest goals. This initiative, launched in June 2023, introduces the Commissioner’s National Priority Review Voucher (CNPV), which significantly reduces the review timeline from the standard 10 to 12 months to as little as one to two months.

Among the initial nine therapies are a gene therapy targeting hearing loss and a drug designed to assist individuals in quitting e-cigarettes and vaping. Each selected product has been identified as addressing critical public health needs or enhancing domestic manufacturing capabilities. The FDA’s Office of New Drugs, which oversees this program, has organized its review process into eight therapeutic areas, with each area subdivided into 27 review divisions responsible for nominating eligible products.

The expedited review process will employ a collaborative, team-based approach reminiscent of a tumor board in oncology. This method involves specialists from various fields convening to determine the best treatment strategies for patients. As part of the CNPV program, the FDA will conduct a one-day meeting to discuss applications once all review steps are completed. While the agency aims to make regulatory decisions within one to two months, it retains the authority to extend the review period if necessary, particularly in cases of incomplete applications or manufacturing issues.

“We prioritize speed, but not at the expense of safety,” stated Martin Makary, FDA Commissioner, during a recent podcast discussing the program. “The same team will make the same decisions, and we are prepared to take additional time if we believe further evaluation is required.”

The CNPV program is designed to encourage the development of products that address significant unmet public health needs, bolster domestic manufacturing as a matter of national security, and ensure equitable drug pricing to enhance accessibility.

Among the therapies selected are:

– **DB-OTO**, a gene therapy by Regeneron Pharmaceuticals, aimed at treating a rare genetic form of hearing loss.
– **RMC-6236** from Revolution Medicines, developed to target pancreatic cancer.
– **Bitopertin** by Disc Medicine, which could potentially become the first disease-modifying treatment for erythropoietic protoporphyria, a rare blood disorder.
– **Cenegermin** (brand name Oxervate) from Italy-based Dompé, which is seeking faster approval for an intranasal formulation to treat non-arteritic anterior ischemic optic neuropathy.
– A domestic manufacturing initiative for the active pharmaceutical ingredient in ketamine by Phlow, as there are currently no domestic suppliers for this ingredient.

Additional products include the antibiotic Augmentin XR, infertility drug pergoveris from EMD Serono, and teplizumab (brand name Tzhield), a treatment developed by Sanofi for delaying advanced type 1 diabetes. Also included is cytisinicline from Achieve Life Sciences, aimed at aiding smoking and vaping cessation.

The FDA anticipates announcing more recipients of the CNPV in the coming months as it continues to promote innovations that have the potential to address pressing healthcare challenges.