Vertex presents longer-term data that shows durability and consistency of Casgevy
14 Jun 2024
Clinical ResultDrug ApprovalGene Therapy
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Source: FiercePharma
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 for sickle cell disease (SCD) gene therapy Casgevy (exa-cel), long-term data is beginning to show the consistent efficacy and durability of the treatment, which is the first ever to be developed using CRISPR gene editing technology.
Of 39 SCD patients who have received Casgevy with at least 16 months of follow-up, 36 (92%) have been free of vaso-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.
Additionally, of those 39 patients, all but one were free of hospitalization related to VOCs for at least 12 consecutive months. The results are consistent with previously reported primary and secondary endpoints in the trial.
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 at least 16 months of follow-up, 49 (94%) achieved transfusion independence for at least 12 straight months. The mean duration of transfusion independence was 31 months, with one patient reaching 59 months.
Additionally, 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 help bolster 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 (NICE), did not recommend Casgevy for commercialization in its draft guidance. In the U.S., the one-time treatment costs $2.2 million. Europe signed off on Casgevy in February.
“The comprehensive data set presented today for adult and adolescent TDT patients adds to the growing body of evidence for Casgevy and it is important to now ensure the availability of this innovative treatment to patients in the real world,” 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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