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Blueprint Medicines announces approval of AYVAKYT for treatment of ISM patients in Europe

Blueprint Medicines Corporation today announced the European Commission has approved AYVAKYT® (avapritinib) for the treatment of adult patients with indolent systemic mastocytosis (ISM) with moderate to severe symptoms inadequately controlled on symptomatic treatment. AYVAKYT is the first and only approved therapy for people living with ISM in Europe.
Associazione Italiana Mastocitosi Patrizia Marcis Georg Pirmin Meyer European Union International At Blueprint Medicines Committee For Medicinal Products Human Use

Vertex Receives CHMP Positive Opinion for the First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia

 - If approved by the European Commission, patients 12 years of age and older with severe sickle cell disease or transfusion-dependent beta thalassemia, for whom hematopoietic stem cell...
United States City Of United Kingdom Saudi Arabia Great Britain Franco Locatelli

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Apellis Pharma Sinks On Indication Of CHMP's Negative Opinion On Pegcetacoplan

Shares of Apellis Pharmaceuticals, Inc. (APLS) are falling more than 21 percent Thursday morning after the company said it expects the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) to give a negative opinion on its marketing authorization application for Pegcetacoplan to treat geographic atrophy.
Apellis Pharmaceuticals Inc European Medicines Agency Committee For Medicinal Products Human Use Apellis Pharmaceuticals Medicinal Products Human Use

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