Update: 30 September 2023
- FDA plans to hold an advisory committee meeting for exa-cel (date to be confirmed).
- Investigation in gentler conditioning for exa-cel is underway.
- The Institute for Clinical and Economic Review (ICER) determined that lovo-cel will be cost-effective if priced between $1.35M to $2.05M. ICER has made recommendations to encourage companies to set prices toward lower end of this range to facilitate access and affordability across all insurance systems (in the USA).
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 8 December 2023.
• 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 35 patients with SCD who had received exa-cel at the time of the analysis, 17 patients were evaluable for the primary and key secondary endpoint at the time of the data cut.
• 16/17 (94.1%) were VOC-free for at least 12 consecutive months
• Mean duration of VOC-free was 18.7 months, with a maximum of 36.5 months.
• 17/17 (100%) did not need hospitalisation for VOCs for at least 12 consecutive months
• All patients who received exa-cel, mean fetal hemoglobin was more than 30% of total hemoglobin by Month 3 and was then maintained at approximately 40.0% through follow-up, with pancellular distribution.
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.