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Rare Diseases: BioMarin Sells FDA Priority Review Voucher for $67.5 Million

July 31, 2014

BioMarin Pharmaceutical announces on July 30th, that the company has sold the Rare Pediatric Disease Priority Review Voucher (PRV) it receives in February 2014, when the FDA approves BioMarin Pharmaceutical’s orphan drug Vimizim (Elosulfase Alfa) for the treatment of the Lysosomal Storage Disorder (LSD) Mucopolysaccharidosis Type IV A (Morquio A syndrome). BioMarin Pharmaceutical receives $67.5 million from Regeneron Pharmaceuticals and Sanofi, in exchange for the voucher. Regeneron Pharmaceuticals hopes to use the voucher to speed the approval of the company’s experimental cholesterol drug, Alirocumab. Alirocumab lowers low-density lipoprotein cholesterol in patients who have previously tried other treatments. Sanofi and Regeneron expect to submit U.S. and EU regulatory submissions for Alirocumab before year end.

The purpose of the FDA’s Rare Pediatric Disease Priority Review Voucher Program is to “encourage the development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.” FDASIA (FDA Safety and Innovation Act) added Section 529 to the Federal Food, Drug, and Cosmetic Act (FD&C).  This provision defines the criteria for the FDA awarding of priority review vouchers to sponsors of rare pediatric disease product applications (New Drug Application or Biologics License Application). The sponsor receives the voucher upon approval of the rare pediatric disease product application. Pediatric PRVs may be sold or transferred, and there is no limit on the number of times a priority review voucher can be transferred.  The FDA goal for reviewing a drug with Priority Review status is six months from the filing date. FDASIA’s Section 529 can be found here (reference Section 908 of FDASIA, which amends the FD&C Act by adding Section 529).

The CEO of BioMarin Pharmaceutical, Jean-Jacques Bienaimé, says:

“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. We are very pleased that a patient population beyond BioMarin’s will potentially be able to benefit from a faster drug application review process.”     

Vimizim is the first:

•   FDA approved treatment for Morquio A Syndrome

•   Drug to receive the Rare Pediatric Disease Priority Review Voucher.

Morquio A Syndrome is an inherited LSD that is caused by the deficiency of an enzyme, N-acetylgalactosamine-6-sulfate sulfatase or GALNS. Vimizim is to replace the missing GALNS enzyme. The deficiency in GALNS causes problems with bone development, mobility, and growth. There are about 3,000 patients with Morquio A Syndrome in the developed world.

Please Note: “Cambio de color a diferente pHs” by Rubashkyn (Own work) [CC-BY-SA-3.0] | Wikimedia Commons.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

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