FDA extends decision dates on bluebird bio's gene therapy drugs beti-cel and eli-cel by 3 months

Bluebird Bio will have to wait an extra three months to gain potential approvals for its breakthrough gene therapies beti-cel and eli-cel.

The FDA has extended its review period for the lentiviral gene therapies by three months, the company announced on Tuesday. Beti-cel, also known as Zynteglo, now carries an approval target date of August 19, 2022, while the target date for eli-cel has been pushed to September 16.

The extension will allow the FDA to review additional clinical information that bluebird submitted in response to the regulator's requests. The information was deemed a “major amendment,” but does not relate to safety data concerning either treatment.

“Gene therapies are complex, potentially transformative treatment options for those living with severe genetic diseases, and we all share a responsibility to be diligent for patients as we progress this novel field,” Andrew Obenshain, bluebird’s CEO, said in a release.

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Two months ago, the FDA accepted bluebird’s biologics license application for beti-cel for those with beta thalassemia who require regular red blood cell transfusions. Last month, the FDA did the same for eli-cel for patients with cerebral adrenoleukodystrophy. If approved, these would become the first lentiviral vector gene therapies in the United States for patients with severe genetic diseases.

The extended dates are not expected to impact the priority review status of either application or the potential for bluebird bio to be granted priority review vouchers. Both candidates have been granted orphan drug, breakthrough therapy and rare pediatric disease status.

The extension comes as the FDA has put a partial clinical hold on another of bluebird’s gene therapy treatments, lovo-cel, for sickle cell disease. Bluebird, which had said it plans to submit its application for lovo-cel in the first quarter of next year, will provide an update on that drug when it reports annual results next month.  

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Beti-cel was approved in Europe in June of 2019 but the company and regulators couldn’t come to an agreement on a fair price for the one-and-done therapy. Last year, bluebird pulled beti-cel from Europe.