The U.S. Food and Drug Administration (FDA) has approved expanding the use of the gene therapy "Casgevy" to include children aged two years and older with sickle cell disease accompanied by recurrent vaso-occlusive crises, referred to as "thalassemia".

It was previously approved for patients aged 12 years and older.

The agency explained that the treatment, scientifically known as exagamglogene autotemcel, was previously approved for patients aged 12 years and older. It involves modifying the patient's stem cells using CRISPR technology to stimulate the production of fetal hemoglobin and reduce the formation of sickle red blood cells.

Casgevy is the first treatment based on CRISPR technology to receive FDA approval, having initially been approved in 2023 for patients aged 12 years and older. The new approval represents the first gene therapy specifically authorized for young children with sickle cell disease.

Sickle cell disease is a genetic disorder caused by a gene mutation that distorts red blood cells into a sickle shape instead of the normal round form, impeding their flow through blood vessels and causing severe pain crises, increased risk of infections, chronic anemia, and long-term organ damage.