- The European Commission has approved Agios Pharmaceuticals Inc's AGIO Pyrukynd (mitapivat) for PK deficiency in adult patients.
- Pyrukynd is a first-in-class, oral PK activator and the first approved disease-modifying therapy for patients in the EU with this rare, debilitating, lifelong hemolytic anemia.
- People with PK deficiency suffer from a lifetime of chronic anemia, associated complications, and symptoms.
- Agios has also applied for marketing authorization for Pyrukynd for PK deficiency in adult patients in Great Britain under the European Commission Decision Reliance Procedure (ECDRP) with the Medicines and Healthcare Products Regulatory Agency (MHRA).
- FDA approved Pyrukynd in February 2022 for hemolytic anemia in adults with PK deficiency.
- The EU marketing authorization was based on results from two pivotal studies, ACTIVATE and ACTIVATE-T, conducted in not regularly transfused and regularly transfused adults with PK deficiency, respectively.
- The Phase 3 ACTIVATE trial of mitapivat achieved its primary endpoint and demonstrated a statistically significant increase in hemoglobin in patients with PK deficiency who are not regularly transfused.
- The Phase 3 ACTIVATE-T trial of mitapivat achieved its primary endpoint and demonstrated a statistically significant and clinically meaningful reduction in transfusion burden for regularly transfused patients.
- Price Action: AGIO shares are up 1.44% at $29.15 on the last check Friday.
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