Tebapivat
- Generic Name
- Tebapivat
Agios' Mitapivat Achieves Primary Endpoint in Phase 3 Trial for Children with PK Deficiency
• 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 study met its primary endpoint with 31.6% of mitapivat-treated patients achieving hemoglobin response compared to 0% in the placebo group, with a favorable safety profile.
• Results from both ACTIVATE-Kids and ACTIVATE-KidsT trials position Agios to pursue marketing approval for mitapivat in pediatric PK deficiency patients.
Agios' Pyrukynd Faces FDA Decision for Thalassemia Treatment by September 2025
• 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.
• Pyrukynd, a pyruvate kinase activator, could become the first oral therapy approved for all thalassemia subtypes, addressing a significant unmet need for patients with limited treatment options.
• Agios completed enrollment for the Phase 3 RISE UP study of mitapivat in sickle cell disease, with topline results expected in late 2025 and a potential U.S. commercial launch in 2026.
Agios Pharmaceuticals Reports Strong Q3 2024 Driven by Vorasidenib Milestone and Mitapivat Progress
• 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.
• The company received $1.1 billion in milestone payments following FDA approval of vorasidenib, which will support future launches and pipeline expansion.
• Phase 3 data for Mitapivat in Thalassemia and Pyruvate Kinase Deficiency (PKD) were positive, and enrollment for the Phase 3 rise up study for sickle cell disease is complete.
• Agios received FDA orphan drug designation for Tebapivat for myelodysplastic syndromes (MDS), highlighting its potential in addressing unmet needs.
Agios' Tebapivat Receives FDA Orphan Drug Designation for Myelodysplastic Syndromes
• Agios Pharmaceuticals' tebapivat (AG-946) has been granted FDA orphan drug designation for treating myelodysplastic syndromes (MDS).
• The designation provides incentives like tax credits and potential market exclusivity for rare disease treatments.
• Tebapivat aims to be the first oral therapy for anemia in lower-risk MDS, affecting 75,000-80,000 patients in the U.S. and EU5.
• Agios has completed a Phase 2a study and is initiating a Phase 2b study of tebapivat in lower-risk MDS.
Drug Development Updates
Stay informed with timely notifications on clinical trials, regulatory changes, and research advancements related to this medication.
© Copyright 2025. All Rights Reserved by MedPath