Bluebird bio has received word from the FDA that its gene therapy candidate, lovotibeglogene autotemcel (lovo-cel) for sickle cell disease (SCD), won’t undergo an advisory committee review. This development was made public by the company on Wednesday.
The Biologics License Application (BLA) for Lovo-cel is currently in the regulatory assessment phase, with the FDA’s decision anticipated by December 20.
The Massachusetts-headquartered biotech company proposes lovo-cel as a one-time treatment for patients 12 years and older with a history of vaso-occlusive events due to SCD. Bluebird’s BLA, which was granted Priority Review by the FDA in June 2023, encompasses efficacy results from the Phase I/II HGB-206 study involving 36 patients and two patients from the Phase III HGB-210 study.
Safety data in the BLA covers information from 50 patients throughout the entire development process of lovo-cel. Additionally, the FDA has previously recognized Lovo-cel with designations such as Orphan Drug, Fast Track, Regenerative Medicine Advanced Therapy, and Rare Pediatric Disease.
Andrew Obenshain, bluebird’s CEO, emphasized, “Lovo-cel stands as the most extensively researched gene therapy for SCD.” The therapy operates by introducing a functional variant of the β-globin gene, which, when mutated, leads to SCD symptoms. Lovo-cel provides the gene directly to a patient’s hematopoietic stem cells, allowing them to produce anti-sickling hemoglobin.
Furthermore, bluebird’s R&D program also includes the completed Phase I/II HGB-205 trial and the ongoing long-term follow-up LTF-307 study.
Obenshain pointed out during an investor call that the FDA’s endorsement of lovo-cel is a cornerstone in bluebird’s future business blueprint, with goals set for near-term profitability and sustained growth within half a decade.
As of June 30, 2023, bluebird reported a cash reserve of $291 million, which is projected to support the company until the close of 2024.
Meanwhile, in the competition to launch an SCD gene therapy, CRISPR Therapeutics, in collaboration with Vertex Pharmaceuticals, finalized its BLA submission in April 2023 for its candidate, exagamglogene autotemcel (exa-cel). This therapy modifies a patient’s hematopoietic stem cells to increase the production of fetal hemoglobin. With a decision date set for December 8 by the FDA, exa-cel might emerge as the inaugural CRISPR-based gene-editing therapeutic to get the green light.