{"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":"
\n

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

Bluebird goes 2-for-2 on gene therapies at FDA adcomm with thumbs up for beti-cel – Endpoints News<\/span> Read More »<\/a><\/p>\n","protected":false},"author":1,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"site-sidebar-layout":"default","site-content-layout":"default","ast-main-header-display":"","ast-hfb-above-header-display":"","ast-hfb-below-header-display":"","ast-hfb-mobile-header-display":"","site-post-title":"","ast-breadcrumbs-content":"","ast-featured-img":"","footer-sml-layout":"","theme-transparent-header-meta":"","adv-header-id-meta":"","stick-header-meta":"","header-above-stick-meta":"","header-main-stick-meta":"","header-below-stick-meta":"","spay_email":"","jetpack_publicize_message":"","jetpack_is_tweetstorm":false,"jetpack_publicize_feature_enabled":true},"categories":[8],"tags":[],"jetpack_featured_media_url":"","jetpack_publicize_connections":[],"jetpack_sharing_enabled":true,"fifu_image_url":"https:\/\/endpts.com\/wp-content\/uploads\/2020\/11\/bluebird-bio-social-AP-scaled.jpg","_links":{"self":[{"href":"https:\/\/harchi90.com\/wp-json\/wp\/v2\/posts\/45508"}],"collection":[{"href":"https:\/\/harchi90.com\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/harchi90.com\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/harchi90.com\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/harchi90.com\/wp-json\/wp\/v2\/comments?post=45508"}],"version-history":[{"count":0,"href":"https:\/\/harchi90.com\/wp-json\/wp\/v2\/posts\/45508\/revisions"}],"wp:attachment":[{"href":"https:\/\/harchi90.com\/wp-json\/wp\/v2\/media?parent=45508"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/harchi90.com\/wp-json\/wp\/v2\/categories?post=45508"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/harchi90.com\/wp-json\/wp\/v2\/tags?post=45508"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}