FDA approves Pyrukynd for hemolytic anemia in adults with pyruvate kinase deficiency.- Agios Pharma
Agios Pharmaceuticals announced that the FDA has approved Pyrukynd (mitapivat) in the U.S. for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency, a rare, debilitating, lifelong hemolytic anemia.
The FDA granted approval to Pyrukynd based on results from two pivotal studies, ACTIVATE and ACTIVATE-T, conducted in not regularly transfused and regularly transfused adults with PK deficiency, respectively.
The Phase III ACTIVATE trial of mitapivat achieved its primary endpoint. Pyrukynd demonstrated a statistically significant increase in hemoglobin in patients with PK deficiency who are not regularly transfused. In 40 percent (n=16) of patients randomized to Pyrukynd, they achieved a hemoglobin response, compared to 0 patients randomized to placebo (2-sided p<0.0001). statistically significant improvements compared to placebo were also demonstrated for all pre-specified secondary endpoints, including markers of hemolysis and ineffective erythropoiesis. patients treated with pyrukynd experienced changes in jaundice (difference in ls mean of pyrukynd minus placebo: -0.4), tiredness (difference in ls mean of pyrukynd minus placebo: -1.1) and shortness of breath (difference in ls mean of pyrukynd minus placebo: -0.3), as assessed with the daily pyruvate kinase deficiency diary (pkdd) where lower scores represent less sign symptom severity. serious adverse reactions occurred in 10 percent (n="4)" of patients receiving pyrukynd, including atrial fibrillation, gastroenteritis, rib fracture and musculoskeletal pain, which each occurred in 1 patient. the most common adverse reactions including laboratory abnormalities (at least 10%) in patients with pk deficiency were estrone decreased (males), increased urate, back pain, estradiol decreased (males) and arthralgia.></0.0001).>
The Phase III ACTIVATE-T trial of mitapivat achieved its primary endpoint. Mitapivat demonstrated a statistically significant and clinically meaningful reduction in transfusion burden for patients who are regularly transfused. It showed that 33 percent (n=9) of patients achieved a transfusion reduction response, defined as an at least 33% reduction in transfusion burden in the 24-week fixed dose period compared with individual historical transfusion burden standardized to 24 weeks. In addition, 22 percent (n=6) of patients were transfusion-free during the fixed-dose period. The adverse reactions reported in the ACTIVATE-T study were consistent with those observed in ACTIVATE.
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