FDA clears Casgevy gene therapy for children as young as two with sickle cell disease
The FDA expanded its approval of Casgevy, the CRISPR-based gene therapy from Vertex Pharmaceuticals and CRISPR Therapeutics, to patients aged 2 and older with sickle cell disease. The therapy had previously been cleared only for patients 12 and up, and the expansion makes an estimated several thousand additional children eligible.
Casgevy is a one-time treatment that edits a patient’s own blood stem cells to raise production of fetal hemoglobin, which counteracts the sickling of red blood cells that causes the disease’s painful and organ-damaging crises. In the pediatric data supporting the expansion, the children who were treated went at least a year without a severe vaso-occlusive crisis.
The practical constraints remain significant: the procedure requires chemotherapy to clear existing bone marrow, weeks of hospitalization, and a list price near $2 million, which limits how quickly the expanded label will translate into treated patients. The FDA cleared the change under a priority review pathway, acting within roughly two months of the filing.