In the trial, 33% of patients who received PYRUKYND achieved a pre-determined reduction in red blood cell red transfusion, including 22% of patients who did not require any transfusions.

Drug Trials Snapshots are part of an overall FDA effort to make demographic data more available and transparent. The information in the Snapshots also highlights where the trials were...

Available data from clinical trials of PYRUKYND are insufficient to evaluate for a drug-associated risk of major birth defects, miscarriage or other adverse maternal or fetal outcomes.

Agios Pharmaceuticals, Incorporated (Applicant) submitted this new drug application (NDA) for mitapivat (tradename Pyrukynd), seeking approval for the treatment of adult patients with pyruvate...

ACTIVATE: A 24-week, randomized, placebo-controlled clinical study assessed the Hb response and safety of PYRUKYND in patients with a presence of at least 2 variant alleles in the PKLR gene, of …

Discover the demonstrated safety profile of PYRUKYND® (mitapivat). Please see Important Safety Info, including full Prescribing Info

Jan 20, 2023 · Welcome to the FDA’s Center for Drug Evaluation and Research’s (CDER’s) Drug Trials Snapshots Summary Report Since January 2015, CDER has shared information on the diversity of …

Your application for Pyrukynd was not referred to an FDA advisory committee because the application did not raise significant public health questions on the role of the drug in the treatment of...

Available data from clinical trials of PYRUKYND are insufficient to evaluate for a drug- associated risk of major birth defects, miscarriage or other adverse maternal or fetal outcomes.

Jan 8, 2025 · “We 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 …