As an FDA decision draws closer on bluebird bio’s sickle cell disease gene therapy, the company has lined up a potential sale of a priority review voucher (PRV).
The agreement, worth $103 million, hinges on an FDA approval for bluebird’s lovo-cel, which would come with a rare pediatric disease PRV that the company could then sell. Bluebird expects the FDA to decide on its lovo-cel application by Dec. 20.
Novartis is the other company in the PRV transfer agreement, a bluebird filing shows. If the deal ends up closing, it'd be bluebird's third PRV sale in about a year.
Previously, the company won PRVs with the FDA approvals of Zynteglo and Skysona. Bluebird then sold the vouchers to argenx and Bristol Myers Squibb for a total of $197 million.
This time around, the lovo-cel PRV sale would "provide an important source of non-dilutive capital for bluebird ahead of the anticipated launch," Chief Financial Officer Chris Krawtschuk said in a statement.
Meanwhile, with the Zynteglo and Skysona launches underway, the company has said it expects its cash runway to extend until the middle of 2024. That financial situation sets the stage for a high-stakes FDA decision—and potential rollout—of lovo-cel.
Bluebird says it's confident in the “robustness and maturity” of its FDA application for lovo-cel. The company is seeking approval for its gene therapy to treat sickle cell disease patients 12 years and older.
Even getting the application submitted was an uphill climb after the FDA put the filing on a partial hold in 2021 due to a case of “persistent, nontransfusion-dependent anemia” in one patient following treatment.
That came after the agency asked for “an analytical comparability strategy” to make sure the final drug product matched clinical supplies, which caused about a one-year delay.
The regulatory timing put the therapy practically neck-and-neck with Vertex and CRISPR Therapeutics’ sickle cell prospect, which faces an upcoming FDA advisory committee meeting.