In 2024, a significant new treatment approved by the FDA for certain patients with beta thalassemia is exagamglogene autotemcel, known by the brand name Casgevy™.
For individuals living with transfusion-dependent beta thalassemia, the landscape of treatment options expanded in 2024 with the approval of a groundbreaking gene therapy.
Based on information, including details found on the chop.edu website regarding Gene Therapy for Transfusion-Dependent Beta Thalassemia, the U.S. Food and Drug Administration (FDA) granted approval in January 2024 for exagamglogene autotemcel (exa-cell). This therapy, marketed under the brand name Casgevy™, represents a new approach for patients with this specific form of thalassemia.
Key details about this new treatment include:
- Treatment Name: exagamglogene autotemcel (exa-cell)
- Brand Name: Casgevy™
- Type of Therapy: Gene Therapy
- Indication: Treatment of transfusion-dependent beta thalassemia
- Eligible Patients: Individuals 12 years of age and older
- Regulatory Approval: Approved by the FDA in January 2024
This approval marks a notable advancement, offering a new therapeutic option for eligible patients with transfusion-dependent beta thalassemia.
