FDA approves label update for Agios's PYRUKYND

Published 01/06/2025, 08:22 AM
AGIO
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Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a biopharmaceutical company, announced on Monday that the U.S. Food and Drug Administration (FDA) has approved an update to the prescribing information for its drug PYRUKYND® (mitapivat).

The update includes new details on liver injury risks observed in patients treated with PYRUKYND for a condition different from its initially approved use.

The updated sections of the U.S. Prescribing Information (USPI) for PYRUKYND, which is used to treat hemolytic anemia in adults with pyruvate kinase (PK) deficiency, now contain warnings and precautions about liver injury. This injury was noted in patients with another condition who were given a higher dose than recommended for PK deficiency. Characterized by an onset within the first six months of treatment, the liver injury included significant increases in alanine aminotransferase levels, sometimes exceeding five times the upper limit of normal, with or without jaundice.

Following these observations, all affected patients stopped using PYRUKYND, and their conditions improved after discontinuation of the treatment. The updated warnings advise healthcare providers to conduct liver tests before starting PYRUKYND treatment and to continue monthly testing for the first six months, followed by periodic monitoring as clinically indicated. If significant liver test elevations or injury are suspected, the new guidance recommends interrupting or discontinuing PYRUKYND use.

The adverse reactions section of the USPI also refers to the instances of hepatocellular injury in patients treated for the different condition, directing readers to the updated warnings and precautions.

The news above is based on an SEC filing.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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