FDA approves Pyrukynd for adults with pyruvate kinase deficiency-related hemolytic anemia
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The FDA approved mitapivat for the treatment of hemolytic anemia in adults with pyruvate kinase deficiency.
Pyruvate kinase (PK) deficiency is a rare inherited disorder that causes anemia via the premature destruction of red blood cells.
Mitapivat (Pyrukynd, Agios Pharmaceuticals) is a first-in-class PK activator available as an oral tablet. It is the first FDA-approved disease-modifying therapy for hemolytic anemia caused by PK deficiency, according to an Agios-issued press release.
The FDA considered mitapivat under the its priority review program and previously granted it orphan drug and fast track designations.
The FDA based approval of mitapivat on results from two clinical studies.
Researchers evaluated mitapivat as part of the ACTIVATE trial, a randomized, multicenter, double-blind, placebo-controlled study of 80 adults with PK deficiency who did not receive regular blood transfusions.
The study met its primary endpoint by significantly increasing hemoglobin in patients with PK deficiency, which researchers defined as a 1.5 g/dL or greater increase in hemoglobin concentration at baseline sustained at two or more scheduled assessments. Forty percent of patients receiving mitapivat had a hemoglobin response compared with none receiving placebo (two-sided P < .0001).
The second trial — ACTIVATE-T — was a single-arm study of 27 adults with PK deficiency who received regular blood transfusions.
The study also met its primary endpoint by conferring a significant reduction in transfusion burden for patients, which researchers defined as a 33% or greater reduction in transfusions during the 24-week treatment timeframe compared with the patient’s historical transfusion needs standardized to a 24-week period.
The most common adverse events associated with mitapivat, occurring in 10% or more of patients, included a decrease in estrone and estradiol levels in males, increased urate levels, back pain and arthralgia.
“The successful ACTIVATE and ACTIVATE-T studies demonstrate the impact of mitapivat in significantly improving hemolysis and anemia in PK deficiency,” Hanny Al-Samkari, MD, hematologist and clinical investigator at Mass General Cancer Center and Harvard Medical School and an investigator in both pivotal phase 3 studies of mitapivat, said in a release. “The FDA approval of mitapivat... is an encouraging step forward for these patients that addresses a significant unmet need.”
References:
Agios Pharmaceuticals. Agios announces FDA approval of PYRUKYND (mitapivat) as first disease-modifying therapy for hemolytic anemia in adults with pyruvate kinase deficiency. https://investor.agios.com/news-releases/news-release-details/agios-announces-fda-approval-pyrukyndr-mitapivat-first-disease. Feb. 17, 2022. Accessed Feb. 18, 2022.
U.S. FDA. FDA approves treatment for anemia in adults with rare inherited disorder. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-treatment-anemia-adults-rare-inherited-disorder. Feb. 17, 2022. Accessed Feb. 18, 2022.
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