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(NewsNation) — A new treatment for children aged 2 or older with sickle cell disease has been approved by the Food & Drug Administration (FDA).
In a Wednesday news release, the FDA announced it had approved Casgevy, the first gene therapy for children with the disease.
“Casgevy is a gene therapy consisting of the patient’s own (autologous) hematopoietic (blood) stem cells, administered as a one-time single dose for intravenous infusion,” the release noted.
“Pediatric patients as young as 2 years of age can now access a critical additional treatment option to treat these debilitating, life-threatening diseases,” Karim Mikhail, the acting director of the Center for Biologics Evaluation and Research, wrote.
According to the Centers for Disease Control and Prevention (CDC), sickle cell disease is often found at birth and involves red blood cells getting stuck in small blood vessels, resulting in “pain and other serious complications.” Individuals suffer from low levels of hemoglobin and often require regular blood transfusions.
Before approval, a trial was undertaken in which Casgevy was administered to 15 patients aged 5 to 11. According to the release, “eight of the nine efficacy evaluable patients with TDT achieved transfusion independence for 12 consecutive months.”
“These disorders carry a heavy burden for children and their families, affecting growth, development, and long-term health in profound ways,” Megha Kaushal, acting deputy director of the Office of Therapeutic Products in CBER, said in the release.
Kaushal added that the therapy “gives these children a meaningful chance at a healthier future.”
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