Agios Reports Fourth Quarter and Full Year 2025 Financial Results and Provides Business Update

PYRUKYND® (mitapivat) worldwide net revenues of $54.0 million for full year; AQVESME™ (mitapivat) for thalassemia now available in U.S. following FDA approval

Published on Feb. 12, 2026

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Agios Pharmaceuticals, Inc., a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, announced its financial results and business updates for the fourth quarter and full year 2025. The company reported PYRUKYND® (mitapivat) worldwide net revenues of $54.0 million for the full year, driven by continued commercial focus in pyruvate kinase (PK) deficiency. Additionally, the company's AQVESME™ (mitapivat) for the treatment of anemia in adults with alpha- or beta-thalassemia received FDA approval and is now available in the U.S.

Why it matters

Agios' strong financial performance and pipeline progress demonstrate the company's ability to deliver innovative medicines for rare diseases. The approval and launch of AQVESME provide a new treatment option for the thalassemia community, while the company's focus on advancing its PK activation franchise into additional indications, such as sickle cell disease, highlights its commitment to expanding its rare disease portfolio.

The details

In the fourth quarter of 2025, Agios reported PYRUKYND® (mitapivat) net revenues of $16.0 million in the U.S. and $4.0 million ex-U.S., driven by continued commercial focus in PK deficiency and favorable gross-to-net adjustments. The company also announced that AQVESME™ (mitapivat) was approved by the FDA in December 2025 as the only medicine for the treatment of anemia in adults with alpha- or beta-thalassemia, regardless of transfusion burden, and is now available in the U.S. following the implementation of its Risk Evaluation and Mitigation Strategy (REMS) program. Additionally, Agios reported progress in its sickle cell disease pipeline, including plans to have a pre-sNDA meeting with the FDA for mitapivat in the first quarter of 2026 and the completion of enrollment for the Phase 2 trial of tebapivat, with topline results expected in the second half of 2026.

  • In December 2025, the U.S. Food and Drug Administration (FDA) approved AQVESME.
  • AQVESME is now available in the U.S. following the implementation of its Risk Evaluation and Mitigation Strategy (REMS) program in late January 2026.
  • Agios will have a pre-supplemental New Drug Application (sNDA) meeting with the FDA in the first quarter of 2026 for mitapivat in sickle cell disease.
  • Agios expects to report topline results from the Phase 2 trial of tebapivat in sickle cell disease in the second half of 2026.

The players

Agios Pharmaceuticals, Inc.

A commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases.

Brian Goff

Chief Executive Officer of Agios Pharmaceuticals, Inc.

PYRUKYND® (mitapivat)

A medicine developed by Agios Pharmaceuticals for the treatment of pyruvate kinase (PK) deficiency.

AQVESME™ (mitapivat)

A medicine developed by Agios Pharmaceuticals for the treatment of anemia in adults with alpha- or beta-thalassemia, regardless of transfusion burden.

U.S. Food and Drug Administration (FDA)

The regulatory agency that approved AQVESME for the treatment of anemia in adults with alpha- or beta-thalassemia.

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What they’re saying

“2025 was another year of continued execution across our portfolio, highlighted by the historic U.S. approval of AQVESME – the only medicine approved to treat anemia in adults with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The U.S. launch is off to a strong start, with AQVESME now available and earning an enthusiastic response from the thalassemia community.”

— Brian Goff, Chief Executive Officer, Agios (Agios Pharmaceuticals)

What’s next

The judge in the case will decide on Tuesday whether or not to allow Walker Reed Quinn out on bail.

The takeaway

Agios' strong financial performance and pipeline progress, including the approval and launch of AQVESME for thalassemia and the advancement of its PK activation franchise into sickle cell disease, demonstrate the company's ability to deliver innovative medicines for rare diseases and its commitment to expanding its rare disease portfolio.