FDA To Decide On Agios' Request For Expanded Approval Of PYRUKYND In Thalassemia By September 7.

(RTTNews) - Agios Pharmaceuticals, Inc. (AGIO), on Wednesday, announced that its supplemental New Drug Application seeking approval for the expanded use of PYRUKYND, for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia, has been accepted for review by the FDA.

The supplemental New Drug Application has been accepted for Standard Review, with a decision date set for September 7, 2025.

The sNDA submission is based on positive data from the ENERGIZE and ENERGIZE-T Phase 3 trials, which evaluated the efficacy and safety of mitapivat in thalassemia patients.

The trials showed encouraging results, demonstrating PYRUKYND's potential to address a significant unmet need in this patient population, particularly for those with limited treatment options.

Thalassemia is a rare inherited blood disorder causing chronic anemia, with severe complications like organ damage and stroke.

If approved for the proposed expanded indication, PYRUKYND could offer a much-needed treatment option for thalassemia patients, potentially improving quality of life and reducing the need for transfusions.

PYRUKYND received FDA approval for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency in February 2022. The drug generated product revenue of $26.8 million for the year ended Dec. 31, 2023, and $25.77 million for the nine months ended September 30, 2024.

AGIO closed Tuesday's (Jan. 07, 2025) trading at $35.12, up 4.71 %. In Premarket trading, the stock is up another 0.77% at $35.39