The US Food and Drug Administration (FDA) will not schedule an advisory committee meeting to evaluate lovotibeglogene autotemcel (lovo-cel), an investigational one-time gene therapy designed for individuals aged 12 years and above who have a history of vaso-occlusive events associated with sickle cell disease (SCD).
The decision was recently announced by Bluebird Bio in a press release.
“Lovo-cel is the most deeply studied gene therapy in development for sickle cell disease and represents the third lentiviral vector gene therapy that the Agency has reviewed from Bluebird—giving us great confidence in the robustness and maturity of our BLA package,” said Andrew Obenshain, chief executive officer of Bluebird Bio. “We remain focused on working with the Agency on its review in anticipation of a decision by the end of this year.”
Previously, the FDA granted Priority Review status to the lovo-cel Biologics Licensing Application (BLA), establishing a Prescription Drug User Fee Act goal date of December 20, 2023.
Read more about SCD experimental therapies
The BLA for lovo-cel is based on efficacy data obtained from a cohort of 36 patients in the HGB-206 study group C, with a median follow-up of 32 months, and 2 patients from the HGB-210 study, each with an 18-month follow-up.
In addition, the submission includes safety data from 50 patients treated across the entire lovo-cel program. The safety data set features 6 patients who have been monitored for 6 or more years, constituting the longest follow-up data within any gene therapy program developed for SCD.
Lovo-cel aims to add functional copies of a modified form of the β-globin gene into a patient’s own hematopoietic stem cells. This modification enables the patient’s red blood cells to produce antisickling hemoglobin, thereby reducing the proportion of abnormal hemoglobin S. The ultimate goal of the therapy is to decrease sickled red blood cells, hemolysis, and other disease complications.
Bluebird Bio’s clinical development program for lovo-cel includes the completed phase 1/2 HGB-205, ongoing phase 1/2 HGB-206, and phase 3 HGB-210 studies, along with a long-term safety and efficacy follow-up study (LTF-307) for individuals treated with lovo-cel in Bluebird Bio-sponsored clinical studies.
Lovo-cel has earned several designations from the FDA, including Orphan Drug designation, Fast Track designation, Regenerative Medicine Advanced Therapy designation, and Rare Pediatric Disease designation.
Reference
Bluebird Bio confirms that FDA has communicated that advisory committee meeting will not be scheduled for lovo-cel gene therapy for sickle cell disease. News release. Bluebird Bio, Inc; August 16, 2023.
