Britain’s medicines regulator has approved the world’s first gene therapy treatment for sickle cell disease and thalassemia.
The Medicines and Healthcare Regulatory Agency said Thursday it has approved Casgevy for patients with sickle cell disease and thalassemia who are 12 years and older.
Casgevy is made by Vertex Pharmaceuticals (Europe) Ltd. and CRISPR Therapeutics.
Until now, bone marrow transplants, an extremely difficult procedure associated with very unpleasant side effects, are the only long-term treatment.
The move could provide relief to thousands of people with the crippling disease in Britain.
Britain’s medicines regulator said its decision to approve the gene therapy for sickle cell disease was based on a study of 29 patients, 28 of whom reported no serious pain problems for at least a year after treatment. In the thalassemia study, 39 of 42 patients who received the therapy did not require a red blood cell transfusion for at least a year after treatment.
Both sickle cell disease and thalassemia are caused by errors in the genes that transport hemoglobin, the protein in red blood cells that transports oxygen.
Millions of people around the world have sickle cell disease. It is more common in people from places where malaria is or was common, such as Africa and India, and is also more common in certain ethnic groups, such as people of African, Middle Eastern and Indian descent.
The Casgevy works by targeting the defective gene in a patient’s bone marrow stem cells so that the body can make properly functioning hemoglobin.
A price for the gene therapy treatment in Britain has not yet been set by Vertex Pharmaceuticals and the company is working with health authorities “to secure reimbursement and access for eligible patients as quickly as possible,” the company said.
In the US, the Food and Drug Administration is also investigating Casgevy and the agency is expected to make a decision early next month before considering a new sickle cell gene therapy.
(With input from AP)
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Updated: Nov 16, 2023 5:29 PM IST