Regeneron Pharmaceuticals and Sanofi announced that the companies intend to use a U.S. Food and Drug Administration (FDA) rare pediatric disease priority review voucher in connection with the Biologics License Application (BLA) submission for alirocumab. The priority review voucher entitles the holder to designate a BLA for priority review, which provides for an expedited 6-month review from the filing date instead of the standard 10-month review.
Regeneron Ireland, an indirect, wholly-owned subsidiary of Regeneron Pharmaceuticals, Inc., purchased the voucher from BioMarin GALNS Ltd., a direct, wholly-owned subsidiary of BioMarin Pharmaceutical, Inc., which had received it through the FDA's Rare Pediatric Disease Priority Review Voucher Program. Sanofi and Regeneron will equally share the purchase price of $67.5 million.
"The priority review voucher program was established to provide incentives that would enhance innovation in key areas of unmet patient need," said Ned Braunstein, M.D., Vice President, Regulatory Affairs at Regeneron. "Our decision to acquire and leverage the voucher is clear evidence that this program is a valuable incentive for biopharmaceutical companies."
Sanofi and Regeneron expect to submit U.S. and EU regulatory submissions for alirocumab before year end. Alirocumab is an investigational monoclonal antibody targeting PCSK9 (proprotein convertase subtilisin/kexin type 9) that is being evaluated for its potential to lower low-density lipoprotein cholesterol (LDL-C) in patients who are not at their current LDL-C target with standard lipid-modifying therapies.
The Pediatric PRV was created by the 2012 Food and Drug Administration Safety and Innovation Act (FDASIA) and is intended to encourage the development of treatments for rare pediatric diseases. Companies that receive a voucher may use it or transfer the voucher, including by sale, to other organizations.