Gene therapy focused biotech Bluebird Bio said that it has entered into a definitive agreement to sell its second Rare Pediatric Disease Priority Review Voucher for $95 million to Bristol Myers Squibb.
Photo: Chris Krawtschuk, chief financial officer of Bluebird Bio
Bluebird was granted two Rare Pediatric Disease Priority Review Vouchers (PRVs) upon the U.S. Food and Drug Administration approvals of the gene therapies Zynteglo for the treatment of beta-thalassemia in adult and pediatric patients requiring regular red blood cell transfusions and Skysona for the treatment of early, active cerebral adrenoleukodystrophy, on August 17, 2022, and September 16, 2022 respectively. On December 29, 2022, Bluebird closed the sale of its first PRV for $102 million to Argenx.
“The sale of our second priority review voucher further bolsters our financial position and provides an important source of non-dilutive funding for the company,” said Chris Krawtschuk, chief financial officer, Bluebird Bio. “Strengthening the company’s balance sheet ensures we enter 2023 with significant momentum behind the commercial launches of our two FDA-approved gene therapies, and the opportunity ahead in sickle cell disease.”
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. Under this program, upon approval, the FDA awards priority review vouchers to sponsors of rare pediatric disease product applications that meet certain criteria. The voucher can be redeemed to receive priority review of a subsequent marketing application for a different product. PRVs may be sold or transferred, and there is no limit on the number of times a PRV can be transferred.
Author: Rare Daily Staff
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