Haydar Frangoul, MD, on Landmark Gene Editing Exa-Cel Approval for SCD

"We are extremely excited about the FDA approval for the first-in-human CRISPR/Cas9 gene-edited cells for individuals with sickle cell disease. It is a long time coming... Seeing it approved today is a huge milestone, not only for the individuals with sickle cell disease, but also for the field of gene editing. This is the first gene editing product that will be approved to be given to humans. And I'm hoping that that will open the door for this to be used more widely in other disease conditions in the future.”

On December 8, 2023, the FDA simultaneously approved Vertex Pharmaceuticals' and CRISPR Therapeutics’ autologous gene-edited cell therapy exagamglogene autotemcel (exa-cel), marketed under the name Casgevy, for the treatment of severe sickle cell disease (SCD), and bluebird bio’s lovotibeglogene autotemcel (lovo-cel), marketed as Lyfgenia, also for SCD.

The approvals were shortly followed up by data presentations on the therapies presented at the 2023 American Society of Hematology (ASH) Annual Meeting & Exposition, held December 9-12, in San Diego, California. Data supporting exa-cel's approval, from the CLIMB SCD-121 trial (NCT03745287), were presented by Haydar Frangoul, MD, Medical Director of Pediatric Hematology/Oncology at Sarah Cannon Research Institute and Pediatric Transplant and Cellular Therapy Program at TriStar Centennial.

The CLIMB SCD-121 trial met primary and key secondary endpoints, with exa-cel treatment resulting in early and sustained increases in Hb and HbF leading to elimination of VOCs in 95% of pts, elimination of inpatient hospitalization for VOCs in 100% of pts and improved QOL, with a manageable safety profile. CGTLive spoke with Frangoul to learn more about his reaction to the approvals of exa-cel and lovo-cel.

Click here to read more coverage of the 2023 ASH Meeting.

REFERENCE
FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease. News release. FDA. December 7, 2023. Accessed December 7, 2023. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease