Health Canada Grants Marketing Authorization of First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY® (Exagamglogene Autotemcel), for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia

September 26, 2024: The GANSID and its members in Canada serving the sickle cell and thalassemia communities are pleased with the news of CASGEVY receiving marketing authorization for the treatment of these two disorders.
CASGEVY is Canada’s first CRISPR-based gene-editing therapy to receive Health Canada’s marketing authorization.
“This marketing authorization will improve the visibility of sickle cell and thalassemia disorders in Canada. Access by many to this cure therapy will also reduce the stigma and discrimination experienced by many living with hemoglobinopathies in the country”, advised Lanre Tunji-Ajayi, M.S.M, CEO of the GANSID.

Press release from Vertex Inc.

TORONTO, Sept. 25, 2024 /CNW/ – Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced Health Canada has granted Marketing Authorization for ^PrCASGEVY^® (exagamglogene autotemcel), an autologous genome edited hematopoietic stem cell-based therapy, for the treatment of patients 12 years of age and older with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT). There are an estimated 2,000 patients eligible for CASGEVY in Canada, the majority of whom are living with SCD.

“Today’s marketing authorization for CASGEVY as Canada’s first CRISPR-based gene-editing therapy is a historic moment,” said Michael Siauw, General Manager at Vertex Pharmaceuticals (Canada) Incorporated. “We are excited for the potential of this one-time therapy for eligible sickle cell disease and transfusion-dependent beta thalassemia patients and are working closely with provincial, territorial and federal governments to facilitate access as rapidly as possible.”

This approval is based on the positive interim results from two global and ongoing clinical trials, CLIMB-121 in SCD and CLIMB-111 in TDT. The primary endpoint of being free from severe VOCs for at least 12 consecutive months or transfusion independence for at least 12 consecutive months was met in both trials. The safety profile is generally consistent with myeloablative conditioning with busulfan and hematopoietic stem cell transplant.

“CASGEVY’s approval is an exciting moment for two patient communities that have long awaited an innovative therapy that brings new hope and possibilities for those in need,” said Kevin Kuo, M.D., Hematologist and Clinician Investigator in the Red Blood Cell Disorders Clinic at University Health Network, and Principal Investigator for the CLIMB-131 clinical program.

Vertex has submitted CASGEVY to both Canada’s Drug Agency (CDA-AMC) and the Institut national d’excellence en santé et en services sociaux (INESSS) in Québec for Health Technology Assessments. For more information on the details of our CDA-AMC applications, including key milestones, please visit here for our SCD submission and here for our TDT submission.

The administration of CASGEVY requires specialized experience in stem cell transplantation; therefore, Vertex is working closely with provincial hospitals and health authorities to establish a network of authorized treatment centers (ATCs) to offer CASGEVY.

To read the full article, click here

Health Canada Grants Marketing Authorization of First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY® (Exagamglogene Autotemcel), for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia

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