FDA Accelerates Review Process for Nine Key Therapies

The U.S. Food and Drug Administration (FDA) has initiated a new pilot program aimed at expediting the review process for nine selected therapies that meet specific national interest criteria. This initiative includes a gene therapy for hearing loss, a smoking cessation drug, and an anesthetic with U.S.-manufactured components. Each of these products has been awarded a Commissioner’s National Priority Review Voucher (CNPV), significantly reducing the standard review period from 10-12 months to just one to two months.

The announcement of this program, made in June 2023, clarified the process by which products would be chosen for this expedited review. The FDA’s Office of New Drugs, which comprises eight specialized review offices, has been assigned the task of nominating products that align with the objectives of the CNPV program. Each of the 27 drug review divisions within these offices can recommend products, and pharmaceutical companies may also apply for consideration.

Details of the Selected Therapies and Their Impacts

The therapies selected for this program are poised to address significant public health needs. For instance, Regeneron Pharmaceuticals‘ DB-OTO is a gene therapy that targets a rare genetic form of hearing loss. Revolution Medicines has developed RMC-6236 specifically for pancreatic cancer, while Disc Medicine‘s bitopertin could potentially become the first disease-modifying treatment for the rare blood disorder erythropoietic protoporphyria.

Italy-based Dompé has received a voucher for cenegermin (marketed as Oxervate), an eye drop treatment currently approved for neurotrophic keratitis. Dompé aims to expedite the FDA approval of an intranasal formulation for treating non-arteritic anterior ischemic optic neuropathy, a condition that can lead to vision loss.

In addition to addressing unmet medical needs, some selected products emphasize the importance of domestic manufacturing. For example, Phlow has received a voucher to produce the active pharmaceutical ingredient for ketamine, an anesthetic. Currently, there are no domestic suppliers for this API, highlighting a gap in U.S. manufacturing capabilities.

Other notable recipients include EMD Serono‘s infertility drug pergoveris, Sanofi‘s teplizumab (branded as Tzhield), which has been approved to delay the onset of severe type 1 diabetes, and Achieve Life Sciences‘ cytisinicline, a drug intended for smoking and vaping cessation.

Regulatory Process and Future Outlook

The FDA’s expedited review process will utilize a collaborative, team-based approach resembling tumor board meetings, where specialists from various fields come together to decide on the best treatment options for patients. The FDA emphasized that while the aim is to accelerate reviews, the agency is committed to maintaining safety standards. FDA Commissioner Martin Makary stated, “We like speed, but we don’t like cutting any corners on safety. That is our number one priority.”

Mallika Mundkur, the FDA’s chief medical officer overseeing the CNPV program, reiterated that eligible products must address significant public health needs and domestic manufacturing issues, as well as promote equitable drug pricing. The agency plans to announce additional groups of voucher recipients in the coming months, expanding the reach of this innovative program.

This initiative reflects the FDA’s ongoing efforts to respond to pressing healthcare challenges while ensuring that safety and efficacy remain paramount in the regulatory review process.