FDA Launches Fast-Track Review for Nine Innovative Therapies

The U.S. Food and Drug Administration (FDA) has unveiled the first nine therapies selected for a new pilot program designed to expedite the regulatory review process for products deemed to meet national priority goals. This initiative includes a gene therapy for hearing loss, a smoking cessation drug, and an anesthetic with its main ingredient manufactured in the United States. Each product has been awarded a Commissioner’s National Priority Review Voucher (CNPV), which significantly reduces the standard review time from 10 to 12 months to just one to two months.

The announcement, made in June 2023, clarified how the FDA would select products for the CNPV program. The FDA’s Office of New Drugs, which consists of eight designated review offices based on therapeutic areas, is responsible for nominating eligible products. There are a total of 27 drug review divisions, each tasked with identifying therapies that align with the CNPV program’s objectives. Additionally, pharmaceutical companies can apply for inclusion in the program, with their requests vetted by the relevant review division.

Streamlined Review Process and Safety Assurance

The FDA aims to implement a team-based review approach, similar to the practice of tumor boards in oncology, where specialists collaborate to determine optimal treatment strategies. Once a CNPV-voucher product has completed all required review steps, the FDA will hold a one-day meeting to discuss the application comprehensively. While the expedited timeframe holds promise, the FDA has emphasized that it reserves the right to extend the review period if necessary, particularly in cases of incomplete applications or manufacturing concerns.

“We like speed, but we don’t like cutting any corners on safety,” stated Martin Makary, FDA Commissioner, during a podcast discussing the CNPV recipients. He reiterated that safety remains the agency’s top priority, and the same decision-making processes will apply, even if additional time is needed.

Mallika Mundkur, the FDA’s Chief Medical Officer and the official overseeing the CNPV program, highlighted that eligible products include those addressing significant public health needs, domestic manufacturing for national security, and efforts to equalize drug pricing compared to other countries.

Innovative Therapies Targeting Major Health Issues

Among the selected therapies is Regeneron Pharmaceuticals’ gene therapy, DB-OTO, aimed at a rare genetic form of hearing loss. Revolution Medicines’ RMC-6236 targets pancreatic cancer, while Disc Medicine’s bitopertin could potentially be the first disease-modifying treatment for the rare blood disorder erythropoietic protoporphyria.

The Italy-based company Dompé has received a voucher for its product cenegermin (brand name Oxervate), which is approved for treating neurotrophic keratitis. Dompé is seeking expedited approval for an intranasal formulation to treat non-arteritic anterior ischemic optic neuropathy, a condition that can lead to vision loss.

Domestic manufacturing concerns have also influenced the selection of voucher recipients. Phlow received a voucher for producing the active pharmaceutical ingredient (API) used in the anesthetic ketamine, as there are currently no domestic suppliers for this component. The antibiotic Augmentin XR was also awarded a voucher for domestic manufacturing. Additional recipients include pergoveris, an infertility drug from EMD Serono, teplizumab (brand name Tzhield) from Sanofi for delaying type 1 diabetes, and cytisinicline from Achieve Life Sciences, aimed at assisting with smoking and vaping cessation.

The FDA anticipates announcing another group of CNPV voucher recipients in the upcoming months, indicating a commitment to addressing pressing health issues through innovative therapies.