FDA Approves First Gene Therapy For Young Children With Sickle Cell Disease

The U.S. Food and Drug Administration has approved Casgevy, the first gene therapy for children aged 2 years and older with sickle cell disease.

According to the report, the one-time treatment expands access to an advanced therapy designed to address the underlying cause of the inherited blood disorder, which can lead to severe pain and other serious health complications.

The report said a clinical trial involving 15 children between the ages of 5 and 11 produced encouraging results.

Participants who had relied on regular blood transfusions achieved at least 12 consecutive months without needing further transfusions after receiving the therapy.

Karim Mikhail of the Center for Biologics Evaluation and Research said eligible children can now access the treatment, while Megha Kaushal of the Office of Therapeutic Products described the therapy as offering many young patients a meaningful opportunity for a healthier future through a single intravenous infusion.

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Gene therapy for children with sickle cell disease approved by FDA https://t.co/Az9yf7Ap9L

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tippinsightsTIPP Staff