Efficacy and safety of mitapivat in adults with transfusion-dependent α-thalassaemia or β-thalassaemia (ENERGIZE-T): a double-blind, randomised, multicentre, placebo-controlled, phase 3 trial.

In 30 seconds

The ENERGIZE-T trial evaluated the efficacy and safety of mitapivat in adults with transfusion-dependent α- or β-thalassaemia. This double-blind, randomised, placebo-controlled phase 3 trial involved 258 patients. Mitapivat led to a transfusion reduction response in 30% of patients compared to 13% in the placebo group, with an adjusted difference of 18 percentage points (95% CI 8-27; p=0.0003).

Key findings

• 30% of patients in the mitapivat group achieved a transfusion reduction response compared to 13% in the placebo group.
• The adjusted difference in transfusion reduction response was 18 percentage points (95% CI 8-27).
• Adverse events occurred in 90% of patients treated with mitapivat, with headache and upper respiratory tract infection being the most common.
• Serious adverse events were reported in 11% of mitapivat patients and 15% of placebo patients.

Why it matters

This study highlights mitapivat as a potential first oral disease-modifying therapy for adults with transfusion-dependent thalassaemia, addressing a significant unmet need in this patient population by reducing transfusion requirements.