The U.S. Food and Drug Administration (FDA) has unveiled the initial nine products selected for its new program aimed at accelerating the regulatory review of therapies that serve national interests. This initiative, announced in June, includes a gene therapy for hearing loss, a smoking cessation drug, and a domestically manufactured anesthetic. Each of these products has been awarded a Commissioner’s National Priority Review Voucher (CNPV), which significantly shortens the standard review timeline from 10 to 12 months down to just one to two months.
The FDA’s Office of New Drugs has established a structured approach for selecting products for this program. Each of the 27 drug review divisions within the office is tasked with nominating therapies that align with the goals of the CNPV initiative. Additionally, pharmaceutical companies can apply for consideration, with their requests being evaluated by the relevant review division. This collaborative effort is designed to expedite the review process while maintaining rigorous safety standards.
During a recent podcast, FDA Commissioner Martin Makary emphasized the agency’s commitment to safety, stating, “We like speed, but we don’t like cutting any corners on safety. That is our number one priority throughout this.” The FDA reserves the right to extend the review period if necessary, particularly in cases of incomplete applications or manufacturing violations.
Selected Products Addressing Key Health Needs
Among the products selected, Regeneron Pharmaceuticals’s DB-OTO is a promising gene therapy targeting a rare genetic form of hearing loss. Another notable entry is Revolution Medicines’ RMC-6236, which has been developed for pancreatic cancer. Disc Medicine’s bitopertin aims to become the first disease-modifying treatment for the rare blood disorder known as erythropoietic protoporphyria.
The program also highlights the importance of domestic manufacturing as a national security concern. Phlow has received a voucher for the domestic production of the active pharmaceutical ingredient in the anesthetic ketamine, addressing a critical gap as there are currently no domestic suppliers for this component. Additionally, the antibiotic Augmentin XR has been included for domestic manufacturing considerations.
Other selected therapies include EMD Serono’s infertility drug pergoveris, Sanofi’s teplizumab (brand name Tzhield), which is approved for delaying the onset of advanced type 1 diabetes, and Achieve Life Sciences’ cytisinicline, a potential drug for smoking and vaping cessation.
Future of the CNPV Program
The FDA has indicated that it will announce additional groups of voucher recipients in the coming months. The CNPV program represents a significant step towards addressing unmet public health needs while fostering domestic pharmaceutical manufacturing. The agency aims to strike a balance between expedited reviews and ensuring the safety and efficacy of the therapies being evaluated.
As the healthcare landscape continues to evolve, initiatives like the CNPV program may play a vital role in enhancing patient access to innovative treatments and improving public health outcomes across various communities.
