Sign in Subscribe
Cybernet Data Systems Inc.

BioMarin Pharmaceutical Inc. Sells Rare Pediatric Disease Priority Review Voucher for $110M

Strategic Insights

— 2 min read

BioMarin Pharmaceutical Inc. announced the sale of its Rare Pediatric Disease Priority Review Voucher (PRV) to an undisclosed purchaser for a lump sum payment of $110,000,000. The company received the voucher under a Food and Drug Administration (FDA) program intended to encourage the development of treatments for rare pediatric diseases. BioMarin was awarded the voucher when it received approval of VOXZOGO (vosoritide) for Injection, indicated to increase linear growth in pediatric patients with achondroplasia five years of age and older with open epiphyses (growth plates).

The transaction remains subject to customary closing conditions, including anti-trust review. The company plans to direct the proceeds from this voucher sale towards additional investment in its already robust pipeline of investigational therapies for people with genetic diseases. This is the third Priority Review Voucher that BioMarin has received, following the approval of Brineura (cerliponase alfa) and Vimizim (elosulfase alfa).

Key Takeaways:

  • BioMarin sold its Rare Pediatric Disease Priority Review Voucher (PRV) for $110,000,000
  • The company received the voucher under a FDA program intended to encourage the development of treatments for rare pediatric diseases
  • BioMarin was awarded the voucher when it received approval of VOXZOGO (vosoritide) for Injection
  • The transaction remains subject to customary closing conditions, including anti-trust review
  • The company plans to direct the proceeds from this voucher sale towards additional investment in its pipeline of investigational therapies for people with genetic diseases
  • This is the third Priority Review Voucher that BioMarin has received
  • The FDA awarded PRVs to BioMarin when Brineura (cerliponase alfa) and Vimizim (elosulfase alfa) were approved

Statistics:

  • $110,000,000: the lump sum payment for the sale of the Rare Pediatric Disease Priority Review Voucher
  • 3: the number of Priority Review Vouchers awarded to BioMarin
  • 2: the number of FDA-approved products that resulted in the awarding of PRVs to BioMarin (Brineura and Vimizim)
  • 6 months: the FDA goal for reviewing a drug with Priority Review status
  • 10 months: the FDA goal for reviewing a drug under standard review

Sources:

  • BioMarin Pharmaceutical Inc., "BioMarin Announces Sale of Rare Pediatric Disease Priority Review Voucher for $110 Million" (February 9, 2022)
  • BioMarin Pharmaceutical Inc., "BioMarin Announces FDA Approval of VOXZOGO (vosoritide) for Injection for the Treatment of Pediatric Patients with Achondroplasia" (November 2021)
  • United States Food and Drug Administration, "Priority Review" (accessed February 14, 2022)
  • BioMarin Pharmaceutical Inc., "BioMarin Announces FDA Approval of Brineura (cerliponase alfa) for the Treatment of Mucopolysaccharidosis Type IIIA" (January 2020)
  • BioMarin Pharmaceutical Inc., "BioMarin Announces FDA Approval of VIMIZIM (elosulfase alfa) for the Treatment of Mucopolysaccharidosis Type IV" (November 2016)

Read more