The Food and Drug Administration (FDA) has granted Orphan Drug designation to mitapivat (AG-348; Agios Pharmaceuticals) for the treatment of thalassemia.

Mitapivat is a first-in-class, oral, selective small molecule allosteric activator of pyruvate kinase-R enzymes. The designation is based on a phase 2 study that established proof-of-concept for mitapivat in 8 patients with non-transfusion-dependent thalassemia. 

Preliminary study analysis showed that 7 patients achieved a hemoglobin increase of ≥1.0g/dL (primary end point) with a mean hemoglobin increase from baseline of 1.76g/dL (range, 0.9–3.3g/dL) during weeks 4-12. Updated study data will be presented at the 25th European Hematology Association (EHA) Annual Congress on June 11-14, 2020. 

The FDA previously granted Fast Track designation to mitapivat for this indication. The Company is also evaluating mitapivat in patients with pyruvate kinase deficiency in 2 ongoing global, pivotal phase 3 trials (ACTIVATE and ACTIVATE-T). Additionally, mitapivat is being investigated for the treatment of sickle cell disease.

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“Receiving Orphan Drug designation is an important milestone as we continue to advance mitapivat for patients with thalassemia, a serious hemolytic anemia with limited treatment options,” said Chris Bowden, MD, chief medical officer at Agios. “We look forward to presenting updated data from our phase 2 study of mitapivat in both alpha- and beta-thalassemia patients at the virtual European Hematology Association Annual Congress later this week.”

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