Vertex presents longer-term data that shows durability and consistency of Casgevy

14 Jun 2024
www.fiercepharma.com
Clinical ResultDrug ApprovalGene Therapy
At the European Hematology Association (EHA) conference, Vertex presented long-term data showing the durability and effectiveness of Casgevy for patients who have taken the gene therapy for sickle cell disease or transfusion-dependent beta thalassemia.
Six months after Vertex earned a historic FDA approval foVertexle cell disease (SCD) gene therapy Casgevy (exa-cel), long-term data iCasgevyning to show the consistent efficacy and durabilitsickle cell disease whitransfusion-dependent beta thalassemiasing CRISPR gene editing technology.
Of 39 SCD patientVertexhave received CasgeFDAwith at least sickle cell disease (SCD)6 (92%) have bCasgevye exa-cel-occlusive crises (VOCs), the debilitating pain episodes that accompany the disorder, for at least 12 consecutive months. The mean duration for patients to remain VOC-free was 28 months, with one patient reaching 55 months, Vertex said.
AdditiSCDlly, of those 39 patients, aCasgevyone were free of hospitalization related to VOCs for at least 12 cvaso-occlusive crises (VOCs)ts are consistent wpainpreviously reported primary and secondary endpoints in the trial.Vertex
In addition to presenting SCD data Friday at the European Hematology Association (EHA) conference in Madrid, Vertex also provided similar results for patients who have received Casgevy for transfusion-dependent beta thalassemia (TDT). Vertex and CRISPR Therapeutics scored FDA approval in the indication five months ago.
Of 52 TDT patients with atSCDast 16 months of follow-up, 49 (94%) achieved transfusion independence for at leVertex straight months. The mean duration of transfusion independencCasgevy1 montransfusion-dependent beta thalassemia (TDT)VertexCRISPR TherapeuticsFDA
AdditiTDTlly, all 52 patients in the study are now transfusion-free. Two of the three patients who did not initially achieve independence have now been transfusion-free for at least a year. The third patient has progressed into a fourth month of transfusion independence.
In both groups, Vertex said that patients reported “sustained clinically meaningful improvements in their quality of life and functional well-being.” Also, in both groups, levels of the diagnostic biomarker BCL11A were stable over time in bone marrow and peripheral blood, indicating successful editing in the long-term hematopoietic stem cells, Vertex added.
The data should Vertexolster Vertex’s push to gain marketing authorization for Casgevy in the U.K. A month ago, the U.K.’s drug pricing gatekeeper, the National Institute for Health and Care Excellence (NICEBCL11A not recommend Casgevy for commercialization in its draft guidance. In the U.S., the one-time treatment costs $2.2 million. Europe siVertexff on Casgevy in February.
“The comprehensive data set pVertexed today for adult and adolescent TDT patients adds to the growing body of evidence for Casgevy and it is important tNational Institute for Health and Care Excellence (NICE)nt to patients in thCasgevyworld,” Franco Locatelli, M.D., Ph.D., professor of pediatrics at the Catholic University of the Sacred Heart of Rome, said in a release. “With the longest follow-up now more than five years, alongside stable editing and sustained fetal hemoglobin levels, I have conviction in the durable benefit to the patients treated with Casgevy.”
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