No AdComm Meeting for Sickle Cell Therapy Lovo-Cel

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Bluebird's gene-edited cell therapy has a PDUFA date of December 20, 2023.

Bluebird bio has announced that the FDA will not be holding an advisory committee meeting prior to the Prescription Drug User Fee Act (PDUFA) goal date of December 20, 2023, for the company’s gene-edited cell therapy lovotibeglogene autotemcel (lovo-cel) for the treatment of patients with sickle cell disease (SCD) aged 12 or older who have a history of vaso-occlusive events.1

“Lovo-cel is the most deeply studied gene therapy in development for SCD 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,” Andrew Obenshain, chief executive officer, bluebird bio, said in a statement.1 “We remain focused on working with the Agency on its review in anticipation of a decision by the end of this year.”

bluebird bio submitted its biologics license application (BLA) for priority review of lovo-cel in April 2023.2 The submission is based on efficacy data from 36 patients from the ongoing phase 1/2 HGB-206 clinical trial (NCT02140554) and 2 patients in the phase 3 HGB-210 clinical trial (NCT04293185). The patients treated in HGB-206 have a median of 32 months of follow-up while the 2 patients from HGB-210 both have 18 months of follow-up.

READ MORE: ICER Rates Exa-Cel and Lovo-Cel Cost-Effective up to $2 Million

The safety data included in the submission comes from 50 patients treated across multiple clinical trials for lovo-cel; 6 of these patients have at least 6 years of follow-up. The serious adverse events (AEs) related to lovo-cel in this dataset include cases of anemia in 2 patients with alpha-thalassemia and leukemia (not resulting from insertional oncogenesis) in 2 patients. Nonserious AEs related to lovo-cel in this dataset include cases of infusion reactions, namely hot flush and decreased blood pressure, in 2 patients. Three of the 50 patients included in the BLA submission’s dataset have died: 1 patient died from sudden cardiac death and 2 patients died from leukemia.

“The severity of sickle cell disease, and its impact on patients and caregivers, has been underappreciated and overlooked for far too long,” Obenshain said in a statement at that time.“Transformative therapies for this community are long overdue. We are pleased to have satisfied the Agency’s questions about comparability to enable our BLA submission, and to take this important step toward making lovo-cel available for individuals living with SCD.”

REFERENCES
1. 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. https://www.businesswire.com/news/home/20230816191251/en/bluebird-bio-Confirms-That-FDA-Has-Communicated-That-Advisory-Committee-Meeting-Will-Not-Be-Scheduled-for-lovo-cel-Gene-Therapy-for-Sickle-Cell-Disease
2. bluebird bio Submits Biologics License Application (BLA) to FDA for lovotibeglogeneautotemcel (lovo-cel) for Patients with Sickle Cell Disease (SCD) 12 years and Older with a History of Vaso-Occlusive Events. News release. bluebird bio, Inc. April 24, 2023. Accessed April 24, 2023. https://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-submits-biologics-license-application-bla-fda-0
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