{"id":798807,"date":"2025-01-08T07:10:38","date_gmt":"2025-01-08T12:10:38","guid":{"rendered":"https:\/\/www.marketnewsdesk.com\/index.php\/fda-accepts-agios-supplemental-new-drug-application-for-pyrukynd-mitapivat-in-adult-patients-with-non-transfusion-dependent-and-transfusion-dependent-alpha-or-beta-thalassemia\/"},"modified":"2025-01-08T07:10:38","modified_gmt":"2025-01-08T12:10:38","slug":"fda-accepts-agios-supplemental-new-drug-application-for-pyrukynd-mitapivat-in-adult-patients-with-non-transfusion-dependent-and-transfusion-dependent-alpha-or-beta-thalassemia","status":"publish","type":"post","link":"https:\/\/www.marketnewsdesk.com\/index.php\/fda-accepts-agios-supplemental-new-drug-application-for-pyrukynd-mitapivat-in-adult-patients-with-non-transfusion-dependent-and-transfusion-dependent-alpha-or-beta-thalassemia\/","title":{"rendered":"FDA Accepts Agios\u2019 Supplemental New Drug Application for PYRUKYND\u00ae (mitapivat) in Adult Patients with Non-Transfusion-Dependent and Transfusion-Dependent Alpha- or Beta-Thalassemia"},"content":{"rendered":"
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CAMBRIDGE, Mass., Jan. 08, 2025 (GLOBE NEWSWIRE) — Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) accepted the company\u2019s supplemental New Drug Application (sNDA) for PYRUKYND\u00ae (mitapivat) for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The review classification for this application is Standard and the Prescription Drug User Fee Act (PDUFA) goal date is September 7, 2025.<\/p>\n

\u201cThalassemia is a rare, lifelong inherited blood disorder that causes chronic anemia and can lead to severe complications, including organ damage, stroke, and other serious health issues, with patients today having limited or no effective treatment options,\u201d said\u00a0Sarah Gheuens, M.D., Ph.D., chief medical officer and head of R&D at Agios. \u201cWe look forward to collaborating with the FDA in the coming months as they continue to review our application, with the goal of bringing PYRUKYND, a disease-modifying oral medication, to thalassemia patients regardless of their genotype or transfusion needs.\u201d<\/p>\n

The sNDA is based on the results from the ENERGIZE and ENERGIZE-T Phase 3 trials evaluating mitapivat versus placebo in adults with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia, respectively. The ENERGIZE randomized clinical trial results were presented at the European Hematology Association\u00a02024 Hybrid Congress<\/a> in June 2024, and the ENERGIZE-T randomized clinical trial results were presented at the 66\u1d57\u02b0<\/a>\u00a0American Society of Hematology Annual Meeting and Exposition<\/a> in December 2024.<\/p>\n

\n About PYRUKYND<\/strong>
\n \u00ae<\/strong>
\n \u00a0(mitapivat)<\/strong>
\n
\n U.S. <\/strong>
\n INDICATION<\/strong>
\n
PYRUKYND is a pyruvate kinase activator indicated for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency.<\/p>\n

\n U.S. IMPORTANT SAFETY INFORMATION<\/strong>
\n
\n Acute Hemolysis: <\/strong>Acute hemolysis with subsequent anemia has been observed following abrupt interruption or discontinuation of PYRUKYND in a dose-ranging study. Avoid abruptly discontinuing PYRUKYND. Gradually taper the dose of PYRUKYND to discontinue treatment if possible. When discontinuing treatment, monitor patients for signs of acute hemolysis and anemia including jaundice, scleral icterus, dark urine, dizziness, confusion, fatigue, or shortness of breath.<\/p>\n

\n Hepatocellular Injury in Another Condition:<\/strong> In patients with another condition treated with PYRUKYND at a higher dose than that recommended for patients with PK deficiency, liver injury has been observed. These events were characterized by a time to onset within the first 6 months of treatment with peak elevations of alanine aminotransferase of >5\u00d7 upper limit of normal (ULN) with or without jaundice. All patients discontinued treatment with PYRUKYND, and these events improved upon treatment discontinuation.<\/p>\n

Obtain liver tests prior to the initiation of PYRUKYND and monthly thereafter for the first 6 months and as clinically indicated. Interrupt PYRUKYND if clinically significant increases in liver tests are observed or alanine aminotransferase is >5x ULN. Discontinue PYRUKYND if hepatic injury due to PYRUKYND is suspected.<\/p>\n

\n Adverse Reactions: <\/strong>The most common adverse reactions including laboratory abnormalities (\u226510%) in patients with PK deficiency were estrone decreased (males), increased urate, back pain, estradiol decreased (males), and arthralgia.<\/p>\n

\n Drug Interactions: <\/strong>\n <\/p>\n