After a rough takeoff into 2022, beleaguered gene therapy specialist bluebird bio has alighted on a spot of good news, courtesy of prominent U.S. drug cost watchdog ICER.
The Institute for Clinical and Economic Review (ICER) has blessed the estimated $2.1 million price tag for bluebird’s transfusion-dependent beta thalassemia (TDT) gene therapy beti-cel, also known as Zynteglo. The one-and-done treatment is now up for a decision at the FDA by Aug. 19, following a delay disclosed in January.
Despite some lingering questions, current evidence suggests “beti-cel provides net health benefits to patients with TDT, and given the high annual costs of standard care, traditional cost-effectiveness modeling finds that this new treatment meets commonly accepted value thresholds at a cumulative price of $2.1 million," ICER said in its draft report. But that's contingent on treatment being funded "through an outcomes-based contract for patients with sustained transfusion independence,” ICER said.
ICER’s beti-cel review is still cooking, and it could change after an ongoing comment period, Pharmaphorum pointed out.
Ahead of its Aug. action date, beti-cel will also go before an FDA advisory committee in June.
For its review, ICER dug into five beti-cel studies. Eighty-nine percent of patients who got beti-cel in bluebird’s phase 3 trials were able to break free from transfusions, ICER said. Across the company’s phase 1/2 and phase 3 trials, transfusion independence was met and maintained over a median follow-up period of 42 months.
“However, this duration is not long enough to remove uncertainty regarding the durability of effect over a longer time period,” ICER cautioned.
The gene therapy hopeful is linked to mild side effects, too, though few patients suffered serious reactions and no deaths were reported in bluebird’s studies, the watchdog noted.
Even as Bluebird positions its med as a potential cure, many thalassemia patients and their families told ICER they’re likely to stick with their current transfusion and chelation treatments. Chelation drugs remove excess iron from the body.
Meanwhile, ICER’s endorsement bodes well, not just for bluebird, but also for other gene therapy players angling to set prices for their one-off treatments. Zynteglo, for its part, already boasts marketing clearance in the European Union and the United Kingdom, where the drug debuted with a roughly $1.7 million price tag, Pharmaphorum added in its report.
For a small player like bluebird, however, “achieving appropriate value recognition and market access” for Zynteglo in Europe proved “untenable,” CEO Andrew Obenshain said last summer. Faced with a stalemate over fair gene therapy pricing, bluebird in August telegraphed plans to “wind down” its operations in Europe and focus on its business stateside.
Should it win approval in the U.S., meanwhile, beti-cel is looking at a market of roughly 1,000 to 1,500 TDT patients, ICER noted. Some 1.25 million people overall are estimated to carry the genetic defect behind thalassemia, according to the group.
The drug’s fate—plus that of bluebird’s other gene therapy hopefuls lovo-cel and eli-cel—will likely decide whether bluebird soars or crashes.
Last month, the company said it was hurting for cash and voiced “substantial doubt regarding its ability to continue as a going concern.”
The company is eyeing ways to refill its coffers, which could include the sale of priority review vouchers from potential approvals of beti-cel and eli-cel. Eli-cel is up for review in cerebral adrenoleukodystrophy (CALD) by September 16. The med also hit a regulatory traffic jam in January.
In another bid to extend its cash runway into the first half of 2023, bluebird early this month said it will cut 30% of its workforce. There were 518 full-time employees in bluebird’s nest as of Jan. 31, according to the company’s annual report.