The Food and Drug Administration (FDA) has extended the review period for the Biologics License Applications (BLAs) for 2 gene therapies: betibeglogene autotemcel (beti-cel) for patients with beta-thalassemia who require regular red blood cell transfusions and elivaldogene autotemcel (eli-cel) for cerebral adrenoleukodystrophy in patients without a matched sibling donor.
The new Prescription Drug User Fee Act (PDUFA) target dates for beti-cel and eli-cel are August 19, 2022 and September 16, 2022, respectively. The FDA extended the target dates to allow time to review additional clinical information for both applications.
Beti-cel is a one-time gene therapy that works by adding functional copies of a modified form of the β-globin gene into a patient’s own hematopoietic stem cells. The BLA submission included data from the phase 3 Northstar-2 (ClinicalTrials.gov Identifier: NCT02906202) and Northstar-3 (ClinicalTrials.gov Identifier: NCT03207009) studies, along with two phase 1/2 studies (ClinicalTrials.gov Identifier: NCT01745120, NCT02151526) and a long-term follow-up study (ClinicalTrials.gov Identifier: NCT02633943).
Eli-cel is an investigational one-time gene therapy designed to add functional copies of the ABCD1 gene into a patient’s own hematopoietic stem cells, resulting in the production of adrenoleukodystrophy protein. The BLA submission included data from the phase 2/3 Starbeam (ClinicalTrials.gov Identifier: NCT01896102) and phase 3 ALD-104 (ClinicalTrials.gov Identifier: NCT03852498) studies, along with a long-term follow-up study (ClinicalTrials.gov Identifier: NCT02698579).
“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,” said Andrew Obenshain, CEO, bluebird bio. “We look forward to continuing to work with the FDA on its ongoing reviews of beti-cel and eli-cel, and to bringing these therapies to patients with beta-thalassemia and cerebral adrenoleukodystrophy in the US later this year.”
bluebird provides update on FDA review timelines for betibeglogene autotemcel (beti-cel) for beta-thalassemia and elivaldogene autotemcel (eli-cel) for cerebral adrenoleukodystrophy (CALD). News release. bluebird bio, Inc. Accessed January 18, 2022. https://www.businesswire.com/news/home/20220118005544/en/bluebird-Provides-Update-on-FDA-Review-Timelines-for-Betibeglogene-Autotemcel-beti-cel-for-Beta-Thalassemia-and-Elivaldogene-Autotemcel-eli-cel-for-Cerebral-Adrenoleukodystrophy-CALD.