– Prespecified Statistical Criterion for the Primary Endpoint in ACTIVATE-KidsT Was Not Met; Results Were Clinically Meaningful, with Observed Response Rates Higher for Mitapivat than Placebo for the ...

Findings showed 28.1% of patients treated with mitapivat achieved a TRR vs 11.8% of patients who received placebo. A phase 3 trial evaluating mitapivat in pediatric patients with pyruvate kinase ...

– Tebapivat Phase 1 Data in Sickle Cell Disease and Phase 2b Trial-in-progress Update in Lower-risk Myelodysplastic Syndromes will be Presented and Published – – Live and Webcast Investor Event with ...

– ACTIVATE-Kids is the First Study to Demonstrate Efficacy of an Oral Therapy for Children with PK Deficiency Who Are Not Regularly Transfused – – Safety Results Consistent with Safety Profile for ...

Agios Pharmaceuticals AGIO announced that the FDA has approved Aqvesme (mitapivat) for the treatment of adult patients with alpha- or beta-thalassemia. With this approval, Aqvesme becomes the only FDA ...

Approval was based on the phase 3 ENERGIZE and ENERGIZE-T studies evaluating the safety and efficacy of mitapivat in adults with alpha- or beta-thalassemia.

Agios Pharma's shares soar 16% after FDA expands approval for its blood disorder drug, Aqvesme, set for late next month.