Rolling review for exagamglogene autotemcel for the potential treatment of sickle cell disease and transfusion-dependent beta thalassemia
Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics announced that Vertex has concluded discussions with the FDA, and the FDA granted exagamglogene autotemcel (exa-cel) a rolling review for the potential treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT)
Vertex will submit its biologics licensing application (BLA) for exa-cel for rolling review, beginning in November 2022 and expects to complete the submission by the end of Q1 2023.
Vertex previously completed discussions with the European Medicines Agency (EMA) and the Medicines and Healthcare products Regulatory Agency (MHRA) on the data required to support those marketing applications and is on track to submit by the end of 2022.
Based on progress in this program to date, exa-cel has been granted multiple important regulatory designations, including Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease Designations from the FDA for both SCD and TDT. Exa-cel has also been granted Orphan Drug Designation (ODD) from the European Commission, as well as Priority Medicines (PRIME) designation from the EMA, for both SCD and TDT.
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