Agios Publishes P-III (ACTIVATE-T) Study Results of Pyrukynd (mitapivat) for Pyruvate Kinase Deficiency in The Lancet Haematology
Shots:
- The P-III (ACTIVATE-T) study evaluate mitapivat in 27 adults patients with PK deficiency who are regularly transfused
- The study met its 1EPs i.e., 37% of patients achieved a ≥33% reduction in transfusion burden @24wk. fixed dose period; 9 achieved a ≥50% reduction, 22% reached a transfusion-free status & 11% achieved Hb concentrations in the normal range @≥8wks. after a transfusion, improvements in 2 PK deficiency–specific PROs measures. The safety profile was consistent with prior reported data, grade ≥3 (30%) with no serious AEs & 1 patient experienced dose reduction due to AEs
- Pyrukynd was approved in the US for hemolytic anemia in adults with PK deficiency & is also under EMA’s review with an expected regulatory decision in the EU at the end of 2022
Ref: Globenewswire | Image: Agios
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