Today, we are taking a look at the latest regarding beta thalassemia (β-thalassemia) in relation to clinical guidance, FDA approvals, and clinical trials. The guideline featured today is from the Thalassemia International Federation (TIF), while three clinical trials outlined are all sponsored by Vertex Pharmaceuticals and focused on the gene-editing therapy CTX001. 

Most Recent β-Thalassemia Guidelines
Recent FDA Approvals for β-Thalassaemia

Below are the medications and therapies recently approved by the FDA that we anticipate being incorporated into future clinical guidelines: 

  • (Aqvesme) mitapivat
    • Agios Pharmaceuticals
    • December 2025
    • Indicated for the treatment of anemia in adults with alpha- or beta-thalassemia.
  • (Casgevy) exagamglogene autotemcel
    • Vertex
    • January 2024
    • Indicated for the treatment of patients aged 12 years and older with: sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs); transfusion-dependent β-thalassemia (TDT).
  • (Zynteglo) betibeglogene autotemcel
    • Bluebird Bio
    • August 2022
    • Indicated for the treatment of adult and pediatric patients with β-thalassemia who require regular red blood cell (RBC) transfusions.

Ongoing Clinical Trials Related to β-thalassemia

The following are ongoing Phase 3 clinical trials based in the United States that we anticipate may impact future β-thalassemia clinical guidance.

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