Print“Our decision to acquire and leverage the voucher is clear evidence that this program is a valuable incentive for biopharmaceutical companies, says Ned Braunstein of Regeneron.”
BioMarin Pharmaceutical sold the priority review voucher it received earlier this year to Regeneron Pharmaceuticals and partner Sanofi for $67.5 million, which intend to use it to expedite approval of their new cholesterol drug alirocumab. The partners reported positive top-line results from nine late-stage trials of alirocumab in people with hypercholesterolemia and said they would equally share the purchase cost of the priority review voucher.
The FDA priority review voucher program is intended to encourage the development of treatments for rare pediatric diseases. It entitles the holder to designate a new biologic drug for priority review, shortening the review time to six months instead of the standard 10-month review time. BioMarin received the voucher in February with U.S. Food and Drug Administration approval of Vimizim, a biologic treatment for the rare pediatric disease known as Morquio A syndrome.
“Leveraging the sale of the Priority Review Voucher to reinvest in products to treat rare and ultra-rare diseases makes the most sense for BioMarin given our stage of growth,” says Jean-Jacques Bienaimé, CEO of BioMarin.
The Rare Pediatric Disease Priority Review Voucher program was created by the 2012 Food and Drug Administration Safety and Innovation Act. Companies that receive a voucher may use it or transfer the voucher, including by sale, to other organizations. BioMarin is the first company to put a price tag on the voucher by selling it to Regeneron and Sanofi.
“The priority review voucher program was established to provide incentives that would enhance innovation in key areas of unmet patient need,” says Ned Braunstein, vice president of 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 that is being evaluated for its potential to lower low-density lipoprotein cholesterol in patients who are not at their current LDL-C target with standard lipid-modifying therapies.
The FDA also has a parallel program for neglected diseases. In March, Knight Therapeutics was awarded a priority review voucher when the agency approved Impavido for the treatment of the tropical diseases leishmaniasis.
August 01, 2014
http://www.burrillreport.com/article-biomarin_sells_priority_review_voucher_for_67_5_million.html
