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.