Press release
Vertex Receives CHMP Positive Opinion for the First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia
- If approved by the European Commission, patients 12 years of age and older with severe sickle cell disease or transfusion-dependent beta thalassemia, for
About this update from Vertex Pharmaceuticals Incorporated
[{"type":"text","content":"\n - If approved by the European Commission, patients 12 years of age and older with severe sickle cell disease or transfusion-dependent beta thalassemia, for whom hematopoietic stem cell transplantation is appropriate and a human leukocyte antigen matched related donor is not available, would be eligible for treatment -\n\n\n- Approval decision from the European Commission is expected in Q1 2024 -\n\n\n BOSTON--(BUSINESS WIRE)--\nVertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for the conditional approval of CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy, for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).\n\n\nIf approved, exa-cel would be the only genetic therapy for patients in the European Union who are 12 years of age and older with either severe SCD with recurrent vaso-occlusive crises (VOCs) or TDT, for whom hematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen matched related HSC donor is not available. An approval decision by the European Commission is expected in February 2024.\n\n\n“This positive opinion is yet another important regulatory milestone underscoring the potentially transformative benefit of CASGEVY for eligible patients with sickle cell and transfusion-dependent beta thalassemia,” said Nia Tatsis, Ph.D., Executive Vice President and Chief Regulatory and Quality Officer at Vertex.\n\n\n“There is an urgent need for new potentially curative treatments in beta thalassemia and sickle cell disease, as people with these diseases still have a shorter life expectancy than the general population and an impaired quality of life,” said Franco Locatelli, M.D., Ph.D., Principal investigator in the CLIMB-111 and CLIMB-121 studies, Professor of Pediatrics at the Catholic University of the Sacred Heart, Rome, and Director of the Department of Pediatric Hematology and Oncology at the Bambino Gesù Children’s Hospital. “As an investigator, I have witnessed first-hand the transformative impact exa-cel can have on patients’ lives and I eagerly await the approval in the European Union.”\n\n\nAbout Sickle Cell Disease (SCD)\n\n\nSCD is a debilitating, progressi...