PK Deficiency
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CLINICAL NEWS | PYRUKYND® Receives Positive CHMP Opinion For The Treatment of PKD in Adults
PYRUKYND® is a first-in-class, oral PK activator that was recently approved by the U.S. Food and Drug Administration (FDA). If approved by the European Commission (EC), PYRUKYND® will be the first approved disease-modifying therapy…
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EHA2022 | Agios Presents New Data Supporting the Benefits of PYRUKYND®In Adults With PKD
Data were presented at the European Hematology Association (EHA) Annual Congress, hosted virtually and in person in Vienna on June 9-12, 2022. “Following the FDA approval of PYRUKYND® for the treatment…
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PYRUVATE KINASE DEFICIENCY | FDA Approves First Drug For The Rare Inherited Anemia
The US Food and Drug Administration (FDA) has announced the approval of Mitapivat (PYRUKYND®) tablets as treatment for haemolytic anemia in adults with pyruvate kinase (PK) deficiency. The oral PK…
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PYRUVATE KINASE DEFICIENCY | TIF Issues Charter of Priorities For The Global PKD Patient Community
This newly launched document aspires to bring together available information about PKD in an effort to identify gaps, and to make useful imperative suggestions so as to understand what changes…
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MITAPIVAT | FDA Grants Priority Review To New Drug For Pyruvate Kinase Deficiency
The NDA was granted a Priority Review designation and has been given a Prescription Drug User Fee Act (PDUFA) action date of February 17, 2022, accelerating the review time from…
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PYRUVATE KINASE DEFICIENCY | Agios Submits New Drug Application To FDA For Mitapivat
“With this NDA filing, we are poised to deliver the first potentially disease-modifying therapy for people with PK deficiency, a chronic, lifelong haemolytic anaemia characterized by serious complications affecting multiple…
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PK Deficiency Resources Now Available On TIF’s Website
Recognizing the similarities of thalassaemia with other rare anaemias, particularly those affecting red blood cells, (e.g. PKD, SCD), and noting the absence of disease-specific united internationally coordinated patient organizations to…
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