bluebird bio has entered into an advanced agreement to sell a Rare Pediatric Disease Priority Review Voucher (PRV) for $103m, if granted, in connection with the potential approval of lovotibeglogene autotemcel (lovo-cel) for sickle cell disease (SCD).
Launched in 2012 under the US Food and Drug Administration’s (FDA) Safety and Innovation Act, Rare Pediatric Disease PRV’s specifically target and support the development of new drugs and biological products for certain, rare paediatric diseases.
Upon approval, the rare paediatric voucher can be redeemed to review any drug under priority review, or it can be transferred or sold an unlimited number of times.
Following the FDA’s Biologics License Application (BLA) approval for lovo-cel and the PVA, under the terms of the agreement, all rights to the PRV will be transferred to the buyer and bluebird will receive $103m upon closing of the sale.
Lovo-cel is designed to treat the underlying causes of SCD through an additional functional gene that promotes the production of anti-sickling adult haemoglobin.
In June, the FDA granted priority review to bluebird’s application seeking approval for the use of lovo-cel in patients ages 12 and older with SCD and who have a history of vaso-occlusive events, with a prescription drug user fee act goal date set for 20 December 2023.
Affecting around 100,000 people in the US, SCD is a group of inherited red blood cell disorders.
Rarer forms of the genetic condition, including HbSD, HbSE and HbSO, form in people with one haemoglobin S gene and another abnormal type of haemoglobin gene code (D, E or O).
Chris Krawtschuk, chief financial officer at bluebird, said: “The potential sale of a PRV would provide an important source of non-dilutive capital for bluebird ahead of the anticipated launch of lovo-cel.
“As the FDA completes its review of lovo-cel, our team… looks forward to a regulatory decision by the end of this year.”
In January 2023, bluebird sold its second Rare Pediatric Disease PRV for $95m to Bristol Myers Squibb, following the FDA’s approval of two gene therapies: Zynteglo (betibeglogene autotemcel) to treat beta-thalassaemia and Skysona (elivaldogene autotemcel) to treat early, active cerebral adrenoleukodystrophy.