Update: 30 September 2023
- Investigation in gentler conditioning for exa-cel is underway.
Update: 30 June 2023
• BLA application submitted to FDA (3 April 2023) for exa-cel for patients with SCD and TDT. The submission is based on the results of the ongoing Phase 3 studies, CLIMB-111 and CLIMB-121, as well as an ongoing long-term follow-up study, CLIMB-131.
• FDA has accepted Priority Review for BLA submission and set a PDUFA (the date by which the FDA must respond to the application) target date of 30 March 2024.
• Dosing in the Phase 1/2/3 CLIMB-111 and CLIMB-121 studies continues, as does the CLIMB-131 long-term follow-up study in patients 12 years of age and older.
• Two additional Phase 3 studies of exa-cel continue to enroll patients 5 to 11 years of age with TDT or SCD.
Data presented at the 28th EHA Annual Congress (9 – 11 June 2023) in Frankfurt (Germany) showed that:
▪ Of the 48 patients with TDT who had received exa-cel at the time of the analysis, 58.3% have genotypes associated with severe disease, beta-zero/beta-zero or other beta-zero-like severe genotypes.
▪ 27 TDT patients who were evaluable:
o 24/27 (88.9%) achieved the primary endpoint of transfusion independence for at least 12 consecutive months
o Mean duration of transfusion-independence was 20.5 months with a maximum of 40.7 months.
o All patients who received exa-cel, mean total hemoglobin was ≥11g/dL at Month 3 and ≥12g/dL from Month 6 onward.
Update: 31 March 2023
- The Phase 1/2/3 CLIMB-111 and CLIMB-121 studies and the CLIMB-131 long-term follow-up study are ongoing in patients 12 years of age and older.
- Two additional Phase 3 studies of exa-cel in pediatric patients with TDT and SCD continue to enroll patients.