Business
Agios Outlines 2026 Strategic Priorities and Key Milestones to Accelerate Rare Disease Portfolio Growth
AQVESME™ (mitapivat) U.S. commercial launch in thalassemia underway following December 2025 FDA approvalPre-sNDA meeting with FDA for mitapivat in sickle cell
About this update from Agios Pharmaceuticals, Inc.
[{"type":"text","content":"AQVESME™ (mitapivat) U.S. commercial launch in thalassemia underway following December 2025 FDA approvalPre-sNDA meeting with FDA for mitapivat in sickle cell disease anticipated in first quarter of 2026, with planned U.S. regulatory submission to followCompany progressing early- and mid-stage pipeline in multiple high-value indicationsClear path to profitability through the company’s existing commercial presence in thalassemia and PK deficiency, with potential to achieve over $1 billion in peak global sales CAMBRIDGE, Mass., Jan. 12, 2026 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced its 2026 strategic priorities and key milestones anticipated during the year. Members of the company’s management team will present this update at the 44th Annual J.P. Morgan Healthcare Conference on Wednesday, January 14, 2026, at 8:15 a.m. PT / 11:15 a.m. ET. “In 2025, Agios delivered another year of strong and consistent execution across our portfolio, marking meaningful progress toward our goal of becoming a sustainable and diversified rare disease company,” said Brian Goff, Chief Executive Officer, Agios. “Last year culminated in the historic U.S. approval of AQVESME™ (mitapivat), our pyruvate kinase (PK) activator and the only medicine approved to treat anemia in adults with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. This approval brings a new, disease-modifying oral option to people living with this debilitating and deadly rare blood disorder. “Entering 2026, the company is at an important inflection point. We will deliver a high-impact U.S. launch of AQVESME in thalassemia, seek to expand our PK activation franchise into additional high-value indications such as sickle cell disease and lower-risk myelodysplastic syndromes, and advance our promising early-stage pipeline with the potential to further diversify across hematologic and rare diseases. We also remain focused on disciplined capital allocation and operational efficiency to support our long-term sustainability. With strong momentum and a clear roadmap, Agios enters the year positioned to deliver transformative innovation and meaningful impact for patients living with rare diseases,” Mr. Goff add...