Benzinga | Jan 18, 2022 07:14AM EST

Bluebird Bio Announced FDA Has Extended the Review Period for the Biologics Licensing Applications for Its Lentiviral Vector Gene Therapies

bluebird bio, Inc. (NASDAQ:BLUE) today announced that the US Food and Drug Administration (FDA) has extended the review period for the biologics licensing applications (BLA) for its lentiviral vector gene therapies betibeglogene autotemcel (beti-cel) for -thalassemia and elivaldogene autotemcel (eli-cel) for cerebral adrenoleukodystrophy (CALD). The revised PDUFA goal dates for beti-cel and eli-cel are August 19, 2022 and September 16, 2022, respectively.

The FDA extended the PDUFA goal dates for beti-cel and eli-cel to allow time to review additional clinical information previously submitted by the company in response to FDA information requests as part of its ongoing reviews. The information was deemed a major amendment. The extension of the FDA review timeline does not relate to new safety events for either beti-cel or eli-cel.

bluebird's BLA submission for beti-cel for adult, adolescent and pediatric patients with -thalassemia across all genotypes who require regular red blood cell (RBC) transfusions was accepted by the FDA for priority review in November 2021. The FDA accepted the BLA for eli-cel for patients with cerebral adrenoleukodystrophy (CALD) under the age of 18 for priority review in December 2021. If approved, beti-cel and eli-cel would be the first lentiviral vector gene therapies for patients with severe genetic diseases in the United States.

"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."

The extended PDUFA goal dates are not expected to impact the priority review status of either BLA or the potential for bluebird bio to be granted priority review vouchers upon approval of beti-cel and eli-cel in 2022. The FDA previously granted both beti-cel and eli-cel Orphan Drug status, Breakthrough Therapy designation and Rare Pediatric Disease designation.

bluebird also provided an update on the FDA's partial clinical hold for the lovotibeglogene autotemcel (lovo-cel) gene therapy clinical program for patients under the age of 18 with sickle cell disease. Consistent with the FDA's clinical hold process, the company has received written questions from the FDA and is continuing to evaluate what impact, if any, the partial clinical hold may have on its projected Q1 2023 timing for submitting the BLA. bluebird plans to provide an update with its annual results in February.