In a recent announcement, the U.S. Food and Drug Administration (FDA) approved the first gene therapy for children aged 2 years and older with sickle cell disease. This inherited blood disorder changes the shape of red blood cells into a crescent (or sickle) shape, disrupting their flow through blood vessels. The result can be poor oxygen delivery, severe pain, and even organ damage.
Although more prevalent in the Mediterranean, the Middle East, and sub-Saharan Africa, sickle cell disease is also a significant public health problem in the U.S., making access to novel treatments crucial. Casgevy, the gene therapy based on the CRISPR/Cas9 gene-editing system, has been approved for patients aged 12 years and older since 2023, but lacked approval for younger children until now.
“Making this therapy available to younger patients opens a critical window for intervention and gives these children a meaningful chance at a healthier future,” said Megha Kaushal, pediatric hematologist and director of the Office of Therapeutic Products at the Center for Biologics Evaluation and Research (CBER), in an FDA statement.