Vertex Receives Health Canada Marketing Authorization for Sickle Cell Disease Treatment

By Connor Hart

Vertex Pharmaceuticals received marketing authorization from Health Canada for its treatment of sickle cell disease.

The Toronto biotechnology company said on Wednesday that the treatment, called Casgevy, has been granted approval for the treatment of patients ages 12 and up who have sickle cell disease with recurrent vaso-occlusive crises, or with transfusion-dependent beta thalassemia.

An estimated 2,000 patients are eligible for the treatment in Canada, according to the company.

This approval was based on positive interim results from two ongoing clinical trials, both of which met their primary endpoints of users being free from severe vaso-occlusive crises for at least 12 consecutive months, or transfusion independence for at least 12 consecutive months.

"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," said General Manager Michael Siauw.

The company said it has submitted Casgevy to both Canada's Drug Agency, as well as the Institut national d'excellence en santé et en services sociaux in Québec, for health technology assessments.

Write to Connor Hart at connor.hart@wsj.com

(END) Dow Jones Newswires

September 25, 2024 16:37 ET (20:37 GMT)

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