{"id":9762,"date":"2023-08-16T10:21:00","date_gmt":"2023-08-16T14:21:00","guid":{"rendered":"https:\/\/platohealth.ai\/fda-wont-hold-advisory-meeting-for-bluebirds-sickle-cell-gene-therapy\/"},"modified":"2023-08-19T18:18:32","modified_gmt":"2023-08-19T22:18:32","slug":"fda-wont-hold-advisory-meeting-for-bluebirds-sickle-cell-gene-therapy","status":"publish","type":"post","link":"https:\/\/platohealth.ai\/fda-wont-hold-advisory-meeting-for-bluebirds-sickle-cell-gene-therapy\/","title":{"rendered":"FDA won\u2019t hold advisory meeting for Bluebird\u2019s sickle cell gene therapy","gt_translate_keys":[{"key":"rendered","format":"text"}]},"content":{"rendered":"
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Dive Brief:<\/h3>\n

Dive Insight:<\/h3>\n

The FDA\u2019s decision to skip an advisory panel may indicate that agency reviewers are more unified in their views of the Bluebird therapy. Or it could simply indicate a higher comfort level with the type of treatment Bluebird is developing; FDA committees unanimously backed two Bluebird gene therapies<\/a> that are delivered in a similar way in June 2022, and both went on to win approval<\/a>.<\/p>\n

By contrast, Vertex and its partner are vying to market the first therapy based on CRISPR gene editing technology. Vertex CEO Reshma Kewalramani told investors<\/a> on an Aug. 1 call that the decision to hold an advisory panel wasn\u2019t surprising, \u201cgiven the new mechanism of action.\u201d The meeting will offer an opportunity to hear from patients and highlight the \u201ctransformative potential\u201d of exa-cel, she said.<\/p>\n

In addition to the comfort level the FDA has after reviewing previous Bluebird applications, the agency may also be more familiar with the latest data because of a protracted filing process for lovo-cel<\/a>, Stifel analyst Benjamin Burnett said in a note to clients. Even though \u201cregulatory situations are inherently opaque,\u201d Burnett said he still expects Vertex and CRISPR\u2019s option to ultimately win FDA approval.<\/p>\n

About 100,000 people in the U.S. have sickle cell disease, a progressive and debilitating disease that significantly shortens the lifespan of its victims. There are currently no therapies that address the underlying genetic cause of the condition. <\/p>\n