The U.S. Food and Drug Administration approved expanded use of Vertex Pharmaceuticals' NASDAQ:VRTX gene therapy in children as young as two with inherited blood disorders, including sickle cell disease, the first such treatment cleared for this age group.
Casgevy, a one-time treatment made from a patient's own blood stem cells, was previously approved for patients aged 12 and older with sickle cell disease or transfusion-dependent beta thalassemia.
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Sickle cell disease is a painful, inherited blood disorder in which the body makes sickle-shaped hemoglobin, preventing red blood cells from properly carrying oxygen to the body's tissues.
In a trial of children aged five to under 12 with sickle cell disease, all eight evaluable patients had no severe vaso-occlusive crises or painful episodes for at least 12 straight months within the first 24 months of infusion.
In beta thalassemia, eight of nine evaluable children achieved transfusion independence for 12 consecutive months, with a median duration of 20.1 months.
The FDA granted approval to Vertex in 53 days after filing under the Commissioner's National Priority Voucher, its new fast-track program designed to shorten review time for a drug application.
In 2023, the FDA approved Vertex's and Genetix Biotherapeutics' gene therapies for sickle cell disease in patients 12 years and older.
Other long-term treatment options for sickle cell disease include bone marrow transplant, which requires matching donors, and the chemotherapy drug hydroxyurea.