Date: 2017-12-18
Type of information: Commercialisation agreement
Compound: Rare Pediatric Disease Priority Review Voucher (PPRV)
Company: Ultragenyx Pharmaceutical (USA - CA) Novartis (Switzerland)
Therapeutic area: Rare diseases - Genetic 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 December 18, 2017, Ultragenyx Pharmaceutical has entered into a definitive agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) to Novartis for $130,000,000. Ultragenyx was awarded the voucher under a FDA program intended to encourage the development of treatments for rare pediatric diseases. Ultrgenyx received the PRV in November when Mepsevii™ (vestronidase alfa) was approved by the FDA for the treatment of children and adults with mucopolysaccharidosis VII (MPS VII, Sly syndrome).
Financial terms:
- Under the agreement, Ultragenyx will receive a lump sum payment of $130 million upon the closing of the transaction, which is subject to customary closing conditions including antitrust review.
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Is general: Yes
