Two of bluebird bio’s gene therapies candidates, key to its future viability, were up for review by the US Food and Drug Administration’s (FDA) Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) on Thursday and Friday of last week.
The independent experts unanimously endorsed bluebird’s elivaldogene autotemcel (eli-cel) for cerebral adrenoleukodystrophy (CALD) on June 9. The panel voted 15 to 0 on the question of whether the benefits of eli-cel outweigh the risks, for the treatment of any sub-population of children with early active cerebral adrenoleukodystrophy (CALD).
The committee’s recommendation is based on the Biologics License Application (BLA) for that treatment, which is currently under priority review by the FDA, with a PDUFA goal date set for September 16, 2022.
The FDA advisors also unanimously backed the company’s betibeglogene autotemcel (beti-cel) gene therapy for people with beta-thalassemia who require regular red blood cell transfusions.
On Friday June 10, they voted 13 to 0 on the question of whether the benefits of beti-cel outweigh the risks for the treatment of people with transfusion-dependent beta-thalassemia.
The FDA verdict on beti-cel’s BLA is due by August 19.
While the FDA does not have to follow the recommendations of the committee, the fact that the decisions are unanimous could be clearly in bluebird’s favor.
Financial challenges
The developments are timely for the gene therapy player given that in April it announced a restructuring drive, in that it was cutting staffing numbers by nearly 30% in its goal to generate US$160m in cost savings over the next two years. That revelation followed on from the biotech's warning in March that its financial position raised “substantial doubt” about its ability to continue as a going concern.
Bluebird bio has faced a number of “unexpected hurdles” in recent times in its bid to get approval for its investigational therapies.
In December 2021, the FDA paused a trial of its gene therapy candidate - lovo-cel - for sickle cell disease patients under the age of 18, while, in January this year, the US regulatory body extended the review period for the BLAs for beti-cel and eli-cel.
It also had challenges selling beti-cel in Europe due to pricing issues; the therapy is licensed as Zynteglo in the EU and the UK.
CEO Andrew Obenshain, on a call in April, noted the combination of those setbacks plus a “tough biotech market” has taken some traditional financing off the table in the near term. “We’re optimistic that these options may be viable sources of funding in the future, but we recognize the need for action today.”
Bluebird said then that it also expects to submit a BLA for lovo-cel in Q1 2023.
