Sanofi and Regeneron Pharmaceuticals, Inc. intend to use a US Food and Drug Administration (FDA) rare pediatric disease priority review voucher in connection with their biologics license application (BLA) submission for alirocumab. Alirocumab is an investigational monoclonal antibody targeting PCSK9 (proprotein convertase subtilisin/kexin type 9), which 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 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. Sanofi and Regeneron expect to submit US and EU regulatory submissions for alirocumab before the end of the year.

The Pediatric PRV was created by the 2012 Food and Drug Administration Safety and Innovation Act 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.

Source: Sanofi