Collaborators Sanofi and Regeneron announced that they plan to leverage a U.S. Food and Drug Administration (FDA) rare pediatric disease priority review voucher for their Biologics License Application (BLA) for alirocumab. The voucher will qualify the BLA for an expedited 6 month review with the agency instead of the traditional 10 months.
Alirocumab is an investigational monoclonal antibody that targets the proprotein convertase subtilisin/kexin type 9 (PCSK9) gene to lower cholesterol. The companies reported that the Phase III ODYSSEY trials of alirocumab met the primary efficacy endpoint with a greater reduction of low-density lipoprotein cholesterol (LDL-C) versus ezetimibe.
Elias Zerhouni, President of Global R&D at Sanofi, said, “The robust data from these studies in more than 5,000 patients is the basis of our global regulatory submissions, which we expect in the U.S. and EU by year end.” The partners hope to speed up the process using the voucher it purchased from BioMarin Pharmaceuticals for $67.5 million.
The voucher was awarded to BioMarin this year under an incentive program by the FDA to promote drug development aimed at rare pediatric disease. The company received the voucher upon approval of its drug Vimizim for the treatment of rare pediatric condition Morquio A syndrome. The voucher can be used for other conditions aside from orphan pediatric diseases. Regeneron’s subsidiary Regeneron Ireland bought the voucher from BioMarin GALNS.
Ned Braunstein, VP of Regulatory Affairs at Regeneron, said, “The priority review voucher program was established to provide incentives that would enhance innovation in key areas of unmet patient need. Our decision to acquire and leverage the voucher is clear evidence that this program is a valuable incentive for biopharmaceutical companies.”
Rival company Amgen said it intends to file an application for its own PCSK9 inhibitor with U.S. and EU authorities later this year, which may help explain Sanofi and Regeneron’s desire to speed up alirocumab’s FDA review.