Shots:

• The approval was based on the P-III (ACTIVATE) & (ACTIVATE-T) studies to evaluate Pyrukynd vs PBO in patients with hemolytic anemia with PK deficiency. The therapy is expected to be available in the US in ~2wks.
• Both trials met their 1EPs i.e., In (ACTIVATE) trial, patients achieved a hemoglobin response (40% vs 0%), improvements for all pre-specified 2EPs including markers of hemolysis & ineffective erythropoiesis, changes in jaundice, tiredness & shortness of breath
• In (ACTIVATE-T) trial, 33% achieved a transfusion reduction, 22% were transfusion-free. The therapy is also under EMA’s review for PK deficiency while the regulatory decision in the EU is expected at end of 2022

 Ref: Globe Newswire | Image: BioSpace