Date: 2017-11-27
Type of information: Commercialisation agreement
Compound: Rare Pediatric Disease Priority Review Voucher (PRV)
Company: Biomarin Pharmaceutical (USA - CA) undisclosed
Therapeutic area: Rare diseases
Type agreement: commercialisation
Action mechanism:
- The Rare Pediatric Disease Priority Review Voucher Program (PPRV) is a FDA program intended to encourage development of new drug and biological products for prevention and treatment of certain rare pediatric diseases. Under this program, a sponsor who receives an approval for a drug or biologic for a "rare pediatric disease" may qualify for a PRV that can be redeemed to receive a priority review of a subsequent marketing application for a different product. The sponsor receives the PRV upon approval of the rare pediatric disease product application and it can be sold or transferred.
Disease:
Details:
- • On November 27, 2017, BioMarin Pharmaceutical announced that it has entered into a definitive agreement to sell the Rare Pediatric Disease Priority Review Voucher (PRV) it obtained in April of this year for a lump sum payment of $125,000,000. BioMarin was awarded the voucher when it received approval of Brineura® for patients with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2). The transaction remains subject to customary closing conditions, including anti-trust review. BioMarin will direct the proceeds from this voucher sale towards additional investment in its pipeline of products to treat rare and ultra-rare diseases.
- This is the second PRV that BioMarin has sold. In July 2014, BioMarin received $67.5 million from Regeneron Ireland in exchange for a voucher awarded when it received approval of Vimizim® for patients with mucopolysaccharidosis type IVA, also known as Morquio A syndrome.
Financial terms:
- The sale of the PRV will be recorded as a $125 million gain on sale of an intangible asset and will also be associated with approximately $25 million of income tax expense. As a result of the sale, Biomarin will update its GAAP Net Loss guidance by the $100 million net after tax gain, and for full-year 2017, the GAAP Net Loss guidance will be reduced to between $(10)
million and $(30) million. The sale of the PRV is a special item that will be excluded from Non-GAAP Income and consequently the Non-GAAP Income guidance for the full-year 2017 is unchanged at $60 to $80 million.
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