Bluebird goes 2-for-2 on gene therapies at FDA adcomm with thumbs up for beti-cel
For the second straight day, the FDA’s Cellular, Tissue and Gene Therapies adcomm voted unanimously in favor of FDA approving a bluebird bio gene therapy, this time by a 13-0 vote in favor of beti-cel as a potential treatment for a blood disorder known as β-thalassemia for those who require regular blood transfusions.
The second straight unanimous thumbs up opens the potential for two FDA approvals later this summer for bluebird — although the agency on Friday raised some manufacturing concerns for both therapies.
Adcomm members praised the efficacy of the potential gene therapy as “outstanding” and “tremendous,” and that the benefits “definitely outweigh the risks.” The FDA presenter’s final benefit-risk assessment slide made clear that bluebird’s clinical studies demonstrate that 89% of subjects with TDT who received beti-cel have achieved transfusion independence.
Andrew Obenshain, CEO of bluebird, said in a statement:
“Today’s advisory committee recommendation is recognition of the substantial body of clinical data that support beti-cel as a potentially curative treatment option for these patients. We are grateful to the members of the beta-thalassemia community who contributed to today’s discussion and remain committed to working with the FDA as it completes its review of the beti-cel Biologics License Application.”
The committee on Thursday also gave a big thumbs up to bluebird bio’s potential gene therapy for the rare but fatal condition known as cerebral adrenoleukodystrophy (CALD) by a vote of 15-0, despite FDA safety concerns.
If both therapies win approval, the biotech also would win two priority review vouchers (worth up to about $200 million total), which could provide desperate relief for bluebird, which laid off about 30% of its workforce in April.
The agency also raised concerns about the lentiviral vector used with beti-cel because of the cancer cases seen developing in those receiving treatment from two other potential bluebird gene therapies that use a similar lentiviral vector with a different safety profile. But most of the adcomm members found the safety profile of beti-cel to be adequate.
The FDA does not have to follow the advice of its adcomm but it often does.
Questions about a potential $2.1 million price tag for beti-cel also might raise some eyebrows, but nonprofit ICER has called the potential gene therapy cost-effective.