In quick succession, bluebird bio has cashed in a pair of speedy FDA review vouchers for nearly $200 million.
Late this week, gene therapy player bluebird said it sold the second of two rare pediatric disease priority review vouchers (PRV) from the FDA for $95 million. The buyer, according to a bluebird securities filing, was pharma major Bristol Myers Squibb.
It isn’t immediately clear what BMS has in store for the PRV. A spokesperson said BMS has "a broad set of opportunities in our pipeline and we are not providing specifics at this time.”
Bluebird, for its part, scooped up the vouchers with the 2022 U.S. approvals of its gene therapies Zynteglo and Skysona in August and September, respectively. In late November, bluebird sold the first PRV to immunology specialist argenx for a cool $102 million. That sale closed on Dec. 29, 2022, bluebird said in a press release Friday.
The cash marks a big boon for bluebird, whose business was on the brink before its pair of approvals last year.
The move “bolsters our financial position and provides an important source of non-dilutive funding for the company,” bluebird CFO Chris Krawtschuk said in a statement. The voucher sales will help bluebird glide into 2023 “with significant momentum behind the commercial launches of our two FDA-approved gene therapies, and the opportunity ahead in sickle cell disease,” he added.
Less than a year ago—in the aftermath of beta thalassemia treatment Zynteglo’s pull from the European market—bluebird warned that its cash needs had raised “substantial doubt regarding its ability to continue as a going concern.” At the time, bluebird said it would focus on improving "cost efficiencies" and selling the priority review vouchers upon potential gene therapy approvals at the FDA.
Now, thanks to its twin gene therapy nods last year, bluebird’s plan to re-line its coffers has largely been a success.
On the commercial front, bluebird is charging $2.8 million for Zynteglo in beta thalassemia before discounts, while cerebral adrenoleukodystrophy (CALD) therapy Skysona carries a $3 million price tag. Both drugs are intended to be single-dose treatments.
Pricing previously proved a point of contention for Zynteglo in Europe, where bluebird ultimately pulled its gene therapy from the market in 2021.
To soften the blow of Zynteglo’s cost in the U.S., bluebird's access strategy revolves around a one-time upfront payment with the option of a rebate for beta thalassemia patients who don't achieve transfusion independence. That could, in turn, help patients recoup up to 80% of the one-time treatment cost, bluebird Chief Commercial Officer Tom Klima explained in an interview last year.
Bluebird won’t offer an outcomes-based payment for Skysona because the rarity and complexity of CALD makes such an arrangement “extremely challenging to implement for both bluebird and payers,” bluebird CEO Andrew Obenshain told investors during a conference call last September.