bluebird bio has entered into a definitive agreement to sell a Rare Pediatric Disease Priority Review Voucher (PRV) for $102m, the company announced.
The Rare Pediatric Disease PRV programme was created to encourage the development of new drug and biological products for the prevention and treatment of certain rare paediatric diseases.
Under the programme, upon approval, the US Food and Drug Administration (FDA) awards PVSs to sponsors of rare paediatric disease product applications that meet a specific set of criteria. The voucher can be redeemed to have any drug reviewed under priority review, or it can be sold or transferred to another company.
bluebird bio received two PRVs upon the FDA approvals of Zynteglo (betibeglogene autotemcel) for the treatment of beta-thalassaemia (beta-thal) in adult and paediatric patients requiring regular red blood cell transfusions, as well as Skysona (elivaldogene autotemcel) for the treatment of early, active cerebral adrenoleukodystrophy (CALD) earlier this year.
“With the sale of our first PRV, we have significantly strengthened our financial outlook,” said Andrew Obenshain, chief executive officer, bluebird bio.
He continued: “As momentum builds across our business, this non-dilutive capital further bolsters the ongoing launches of our two recently approved gene therapies and the execution of near-term, value-creating milestones, including the planned submission and subsequent FDA review of our biologics licensing application for lovo-cel for sickle cell disease.”
Beta-thal is a rare genetic blood disease caused by a gene defect that impairs the ability of red blood cells to produce haemoglobin. Patients with the most severe form of the disease develop life-threatening anaemia and have to undergo regular blood transfusions, a lengthy process typically needed every two to five weeks.
Zynteglo works by adding functional copies of a modified form of the beta-globin gene into a patient’s own haematopoietic stem cells to allow them to make normal to near normal levels of total haemoglobin without regular red blood cell transfusions.
CALD is a rare, progressive, neurodegenerative disease that primarily affects young children and leads to irreversible loss of neurologic function and death, with nearly half of patients who do not receive treatment dying within five years of symptom onset.
Prior to the approval of Skysona, effective options were limited to allogeneic haematopoietic stem cell transplant. This stabilises neurologic function, but is associated with serious potential complications and mortality that increase in patients without a matched sibling donor.