bluebird bio sells second FDA priority review voucher for $95m

bluebird bio has agreed to sell its second Rare Pediatric Disease Priority Review Voucher (PRV) for $95m, the gene therapy developer announced.

The company’s first voucher was sold for $102m to Dutch biotech Argenx, which expected to use the PRV for a future marketing application for efgartigimod, its first-in-class neonatal Fc receptor blocker.

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.

As part of the programme, upon approval, the US Food and Drug Administration (FDA) awards PRVs to sponsors of rare paediatric disease product applications that meet a specific set of criteria. The voucher can be used for a priority review of any drug, 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 in adult and paediatric patients requiring regular red blood cell transfusions, and Skysona (elivaldogene autotemcel) for the treatment of early, active cerebral adrenoleukodystrophy last year.

The company’s chief financial officer, Chris Krawtschuk, said: “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.

“Strengthening the company’s balance sheet – as we have continued to do through demonstrated fiscal discipline and the sale of both PRVs – 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 (SCD).”

In December 2022, the FDA lifted its partial clinical hold on bluebird bio’s studies evaluating its lovotibeglogene autotemcel (lovo-cel) gene therapy for patients with SCD who are under the age of 18 years.

The clinical studies were placed on a partial hold in December 2021 relating to an investigation by bluebird bio into an adolescent patient with persistent, non-transfusion-dependent anaemia following treatment with lovo-cel.

The company is now resuming the enrolment and treatment of patients aged between two to 17 years of age and said it remains ‘on track’ to submit a Biologics License Application to the FDA for lovo-cel in the first quarter of 2023, having already completed one study in its lovo-cel clinical development programme.