RARE Daily

BioMarin Sells Priority Review Voucher for $110 Million

February 9, 2022

BioMarin Pharmaceutical said that it has sold its rare pediatric disease priority review voucher to an unnamed buyer for $110 million.

The FDA awarded the voucher to the company in November 2021 with the U.S. regulatory approval of Voxzogo (vosoritide) for injection, indicated to increase linear growth in pediatric patients with achondroplasia five years of age and older with open growth plates.

The transaction remains subject to customary closing conditions, including anti-trust review. This is the third priority review voucher that BioMarin has received. The FDA also awarded PRVs to the company when Brineura (cerliponase alfa) and Vimizim (elosulfase alfa) were approved. 

The Rare Pediatric Disease Priority Review Voucher Program is intended to encourage development of new drug and biological products for the prevention and treatment of certain rare pediatric diseases. A PRV is issued to the sponsor of a rare pediatric disease product application and entitles the holder to priority review of a single New Drug Application or Biologics License Application. The sponsor receives the voucher upon approval of the rare pediatric disease product application. PRVs may be sold or transferred, and there is no limit on the number of times a PRV can be transferred. 

Prior to approval, each drug marketed in the United States must go through a detailed FDA review process.  In 1992, under the Prescription Drug User Act (PDUFA), FDA agreed to specific goals for improving the drug review time and created a two-tiered system of review times, Standard Review and Priority Review. A Priority Review designation is given to drugs that provide a significant improvement in the safety or effectiveness of the treatment, prevention, or diagnosis of a serious condition. The FDA goal for reviewing a drug with Priority Review status is six months from the time the application is filed by the FDA, compared to 10 months under standard review.

Author: Rare Daily Staff

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