Date: 2015-07-06
Type of information: Commercialisation agreement
Compound: Rare Pediatric Disease Priority Review Voucher (PPRV)
Company: Retrophin (USA - CA) Sanofi (France)
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 July 6, 2015, Retrophin announced the closing of the agreement to sell its Rare Pediatric Disease Priority Review Voucher to Sanofi. The Rare Pediatric Disease Priority Review Voucher Program is intended to encourage development of new drug and biological products for prevention and treatment of certain rare pediatric diseases. A voucher may be issued to the sponsor of a rare pediatric disease product application and would entitle the holder to priority review of a single New Drug Application or Biologics License Application, which reduces the target review time and could lead to an expedited approval. The sponsor receives the voucher upon approval of the rare pediatric disease product application.
- • On May 27, 2015, Retrophin announced an agreement to sell its Rare Pediatric Disease Priority Review Voucher ("Pediatric PRV") to Sanofi. Retrophin received the Pediatric PRV when Cholbam™ was approved by the FDA for the treatment of pediatric and adult patients suffering from bile acid synthesis disorders due to single enzyme defects, and for patients with peroxisomal disorders (including Zellweger spectrum disorders). Upon closing of the transaction, Retrophin will receive a payment of $150 million, followed by two equal installments of $47.5 million in 2016 and 2017. The Company will receive a total consideration of $245 million in cash from Sanofi in exchange for the Pediatric PRV. The voucher was awarded by the FDA under a provision that encourages development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.
Financial terms:
- Under the terms of the agreement, Retrophin has received a payment of $150 million, and will receive two additional payments of $47.5 million in 2016 and 2017.
Latest news:
Is general: Yes
