Rolling BLAs to the FDA for exa-cel for the treatment of sickle cell disease and transfusion-dependent beta thalassemia are completed

The BLAs include requests for Priority Review, which if granted, would shorten the FDA’s review of the application to eight months from the time of submission versus a standard review timeline of 12 months.

These submissions are supported by results from the ongoing Phase III studies, CLIMB-111 and CLIMB-121, as well as an ongoing long-term follow-up study, CLIMB-131. Data from the Phase III studies were most recently presented at the American Society of Hematology (ASH) Annual Meeting and Exposition in December of 2022.

In the U.S., exa-cel has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA for both SCD and TDT.

In Europe, the Marketing Authorization Applications (MAAs) for exa-cel were submitted in December 2022 and validated by the European Medicines Agency (EMA) and the Medicines and Healthcare products Regulatory Agency (MHRA) in January 2023. In the EU, exa-cel has been granted Orphan Drug Designation from the European Commission, as well as Priority Medicines (PRIME) designation from the EMA, for both SCD and TDT. In the U.K., exa-cel has also been granted an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) from the MHRA.