{"id":45508,"date":"2022-06-10T22:33:58","date_gmt":"2022-06-10T22:33:58","guid":{"rendered":"https:\/\/harchi90.com\/bluebird-goes-2-for-2-on-gene-therapies-at-fda-adcomm-with-thumbs-up-for-beti-cel-endpoints-news\/"},"modified":"2022-06-10T22:33:58","modified_gmt":"2022-06-10T22:33:58","slug":"bluebird-goes-2-for-2-on-gene-therapies-at-fda-adcomm-with-thumbs-up-for-beti-cel-endpoints-news","status":"publish","type":"post","link":"https:\/\/harchi90.com\/bluebird-goes-2-for-2-on-gene-therapies-at-fda-adcomm-with-thumbs-up-for-beti-cel-endpoints-news\/","title":{"rendered":"Bluebird goes 2-for-2 on gene therapies at FDA adcomm with thumbs up for beti-cel – Endpoints News"},"content":{"rendered":"
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 \u03b2-thalassemia for those who require regular blood transfusions.<\/p>\n
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.<\/p>\n
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.<\/p>\n
Andrew Obenshain, CEO of bluebird, said in a statement:<\/p>\n
\n“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. “<\/p>\n<\/blockquote>\n
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.<\/p>\n
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.<\/p>\n
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.<\/p>\n
The FDA does not have to follow the advice of its adcomm but it often does.<\/p>\n
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.<\/p>\n<\/p><\/div>\n","protected":false},"excerpt":{"rendered":"
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 \u03b2-thalassemia for those who require regular blood transfusions. The second …<\/p>\n