The CRISPR-based medicine Casgevy, approved in December by the US Food and Drug Administration (FDA) to treat sickle cell disease, has been given the green light to also treat transfusion-dependent beta thalassemia in people 12 and older.
Like sickle cell, beta thalassemia is an inherited blood disorder, reports CNN.
To make Casgevy, a person’s stem cells are genetically modified using the precision gene-editing CRISPR/Cas9 technique. The modified cells are then transplanted back into the body, where they grow and multiply and increase the production of haemoglobin – decreasing symptoms.
The treatment lists for $2.2m for both sickle cell disease and beta thalassemia.
In the past, beta thalassemia has been managed with frequent blood transfusions. However, this can lead to iron overload syndrome, which must then be managed with other treatments.
There are at least 1 200 people in the US with thalassemia severe enough that it must be managed with blood transfusions.
CNN article – FDA clears first CRISPR treatment for a second disease, beta thalassemia (Open access)
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