Julie Kanter, MD, on Preparing for Anticipated Approval of Lovo-Cel for Sickle Cell

Commentary
Video

The director of the Adult Sickle Cell Clinic and associate professor at University of Alabama Birmingham discussed the anticipated approval of the first gene therapy for SCD.

“I do support [lovo-cel's] approval. I think it's going to be very complicated, because we need to support sickle cell disease centers in delivering this product, as well as the product itself, which are sort of 2 different price streams. And I think it'll be a little bit complicated as we wander into this new territory of high-priced therapies. But I think the patients that I have taken care of would tell you it is absolutely worth it. It is truly a transformative therapy.”

The sickle cell disease (SCD) community is eagerly anticipating December 20th's Prescription Drug User Fee Act (PDUFA) date for lovotibeglogene autotemcel (lovo-cel; bluebird bio), the first gene therapy to be potentially approved for the treatment of SCD. Although the approval would represent a big step forward in the treatment landscape of SCD, there is more work to be done in preparing the field for gene therapy as well as solving lingering access issues for patients.

CGTLive recently spoke with Julie Kanter, MD, director, Adult Sickle Cell Clinic and associate professor, hematology and oncology, University of Alabama Birmingham, investigator on multiple of lovo-cel's clinical trials, including the HGB-206 (NCT02140554) and HGB-210 (NCT04293185) trials, leading up to lovo-cel's PDUFA date. Kanter discussed her anticipation for the upcoming potential approval but stressed that it will be a complicated process to integrate the new mode of treatment into patients’ regimens. She touched on some concerns such as learning how to manage and transition patients to potentially receiving gene therapy.

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. August 16, 2023. 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
Recent Videos
Michael Flanagan, PhD, chief scientific officer at Avidity
David Barrett, JD, the chief executive officer of ASGCT
David-Alexandre C. Gros, MD, Eledon’s chief executive officer
David Barrett, JD, the chief executive officer of ASGCT
Alfred L. Garfall, MD, MS, associate professor of medicine (hematology-oncology) and director, Autologous Hematopoietic Cell Transplantation, Cell Therapy and Transplant Program, Hospital of the University of Pennsylvania; and section chief, Multiple Myeloma, Division of Hematology/Oncology, Department of Medicine, Perelman School of Medicine, University of Pennsylvania,
Reena Sharma, MD, an adult metabolic consultant at Salford Royal Hospital
Nirav Shah, MD, MSHP, associate professor of medicine, at the Medical College of Wisconsin
Bhagirathbhai R. Dholaria, MD, an associate professor of medicine in malignant hematology & stem cell transplantation at Vanderbilt University Medical Center
Reena Sharma, MD, an adult metabolic consultant at Salford Royal Hospital
Mark Hamilton, MD, PhD, a hematology-oncology and bone marrow transplant (BMT) cell therapy fellow at Stanford University
© 2025 MJH Life Sciences

All rights reserved.