Britain approves world’s first gene therapy Casgevy for sickle cell disease and thalassemia

The Medicines and Healthcare Regulatory Agency on Thursday said that it approved Casgevy for patients with sickle cell disease and thalassemia who are 12 years old and over.

Casgevy is made by Vertex Pharmaceuticals (Europe) Ltd. and CRISPR Therapeutics. 

To date, bone marrow transplants, an extremely arduous procedure that come with very unpleasant side effects, have been the only long-lasting treatment.

The move can offer relief to thousands of people with the crippling disease in the UK.

The UK drug regulator said that its decision to authorize the gene therapy for sickle cell disease was based on a study done on 29 patients, of whom 28 reported having no severe pain problems for at least one year after being treated. In the study for thalassemia, 39 out of 42 patients who got the therapy did not need a red blood cell transfusion for at least a year after the treatment.

Both sickle cell disease and thalassemia are caused by errors in the genes that carry hemoglobin, the protein in red blood cells that carry oxygen.

Millions of people around the world have sickle cell disease. It occurs more often among people from places where malaria is or was common, like 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 been yet established by Vertex Pharmaceuticals and it is working with health authorities “to secure reimbursement and access for eligible patients as quickly as possible”, said the company.

In the US, the Food and Drug Administration is also reviewing Casgevy and the agency is expected to take a decision early next month, before considering another sickle cell gene therapy. 

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Updated: 16 Nov 2023, 05:29 PM IST

The Medicines and Healthcare Regulatory Agency on Thursday said that it approved Casgevy for patients with sickle cell disease and thalassemia who are 12 years old and over.

Casgevy is made by Vertex Pharmaceuticals (Europe) Ltd. and CRISPR Therapeutics. 

To date, bone marrow transplants, an extremely arduous procedure that come with very unpleasant side effects, have been the only long-lasting treatment.

The move can offer relief to thousands of people with the crippling disease in the UK.

The UK drug regulator said that its decision to authorize the gene therapy for sickle cell disease was based on a study done on 29 patients, of whom 28 reported having no severe pain problems for at least one year after being treated. In the study for thalassemia, 39 out of 42 patients who got the therapy did not need a red blood cell transfusion for at least a year after the treatment.

Both sickle cell disease and thalassemia are caused by errors in the genes that carry hemoglobin, the protein in red blood cells that carry oxygen.

Millions of people around the world have sickle cell disease. It occurs more often among people from places where malaria is or was common, like 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 been yet established by Vertex Pharmaceuticals and it is working with health authorities “to secure reimbursement and access for eligible patients as quickly as possible”, said the company.

In the US, the Food and Drug Administration is also reviewing Casgevy and the agency is expected to take a decision early next month, before considering another sickle cell gene therapy. 

Milestone Alert!Livemint tops charts as the fastest growing news website in the world 🌏 Click here to know more.

Updated: 16 Nov 2023, 05:29 PM IST