FDA Approves Agios' Mitapivat As First Disease-Modifying Therapy For Genetic Blood Disorder

The FDA approved Agios Pharmaceuticals Inc's AGIO Pyrukynd (mitapivat) for hemolytic anemia in adults with pyruvate kinase (PK) deficiency.

• Pyruvate kinase deficiency is an inherited metabolic disorder of the enzyme pyruvate kinase that affects red blood cells' survival.
• The company notes that Pyrukynd is a first-in-class, oral PK activator and the first approved disease-modifying therapy for this disease.
• Pyrukynd is expected to be available in the U.S. approximately two weeks after approval. It is also under review by the European Medicines Agency for PK deficiency, with a decision expected by the end of 2022. 
• Related: Agios Presents Mitapivat Long-Term Data On Hemoglobin Response, Transfusion Burden.
• The FDA approval is based on results from ACTIVATE and ACTIVATE-T studies.
• In Phase 3 ACTIVATE trial, 40% of patients randomized to Pyrukynd achieved a hemoglobin response, compared to 0 patients randomized to placebo.
• In Phase 3 ACTIVATE-T trial, mitapivat demonstrated a statistically significant and clinically meaningful reduction in transfusion burden for regularly transfused patients.
• The company expects to initiate ACTIVATE-kids and ACTIVATE-kidsT – in pediatric patients with PK deficiency in mid-2022.
• Price Action: AGIO shares closed 6.75% higher at $31.45 during after-hours trading on Thursday.