FDA approves Pyrukynd for hemolytic anemia in adults

The FDA on Thursday approved Agios Pharmaceuticals' Pyrukynd (mitapivat) for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency, regardless of transfusion status
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The company noted that its oral PK activator is the first approved modifiable therapy for a rare blood disorder

FDA

Pyrukynd (mitapivat) was granted priority review by the FDA last year based on the results of two Phase III trials
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In the ACTIVATE study, Pyrukynd demonstrated a significant increase in hemoglobin (Hb) in PK-deficient patients who received occasional blood transfusions, with 40% of participants achieving an Hb response compared to none in the placebo group

Pyrukynd was also successful in the ACTIVATE-T trial, demonstrating a "significant and clinically meaningful" reduction in transfusion burden in regularly transfused patients
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In this study, 33% of patients achieved transfusion reduction responses above a certain threshold over a 24-week fixed-dose period

Pyrukynd was also successful in the ACTIVATE-T trial, demonstrating a "significant and clinically meaningful" reduction in transfusion burden in regularly transfused patients

Meanwhile, Agios expects to initiate two additional studies of Pyrukynd in mid-2022, ACTIVATE-kids and ACTIVATE-kidsT, in unscheduled and regularly transfused pediatric patients with PK deficiency, respectively
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Pyruvate kinase (PK) deficiency is an autosomal recessive erythrocyte enzyme abnormality that causes chronic hemolysis
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It is the second most common erythrocyte enzyme deficiency, but the most common cause of chronic hemolytic anemia due to erythrocyte enzyme deficiency

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