Mitapivat demonstrated significant efficacy in the ACTIVATE-Kids Phase 3 trial, becoming the first oral therapy to show positive results in children with non-transfusion-dependent PK deficiency.
The FDA has accepted Agios' sNDA for Pyrukynd (mitapivat) to treat adults with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia, setting a PDUFA goal date of September 7, 2025.
Agios Pharmaceuticals reported $9 million in net Pyrukynd revenue for Q3 2024, a 22% increase compared to Q3 2023, alongside a strong cash position of $1.7 billion.