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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 , European Commission , Blueprint Medicines Corporation , Medicines Corporation , Pirmin Meyer , Senior Vice President , Jens Panse , Deputy Director , University Hospital , Medicinal Products , Human Use , Blueprint Medicines ,

Europe Approves Avapritinib for Rare Mast Cell Disease

The drug targets a mast cell mutation that gives rise to indolent systemic mastocytosis, a rare and debilitating blood disorder. ....

European Union , European Commission , Medicinal Products , Human Use , Rare Diseases , Lysosomal Storage Disease , Systemic Mastocytosis , I Cell Disease , Ucolipidosis Type Ii , Nclusion Cell Disease , Ucolipidosis Ii , Ol Ii , Bone Marrow , Edulla Ossea ,

ASH 2023: Phase II case Cogent enough with bezu in mastocytosis? Street skeptical, more data soon

Wall Street’s measure of how Cogent Biosciences Inc.’s KIT D816V inhibitor bezuclastinib (often shortened to bezu) might fare in mastocytosis against U.S. FDA cleared Ayvakit (avapritinib), the tyrosine kinase inhibitor from Blueprint Medicine Corp., caused the former’s stock (NASDAQ:COGT) to tumble, closing Dec. 11 at $4.06, down $4.58, or 53%. ....

San Diego , United States , Cogent Biosciences Inc , American Society Of Hematology , Blueprint Medicine Corp , Wall Street , Cogent Biosciences , American Society , Mast Cell , Cogent Biosciences Inc ,

Targeted therapy reduces markers of disease burden, improves symptoms for patients with nonadvanced system mastocytosis

The targeted therapy bezuclastinib was safe and rapidly reduced markers of disease burden while also improving symptoms for patients with a rare blood disorder called nonadvanced system mastocytosis, according to results of the Phase II SUMMIT trial reported by researchers at The University of Texas MD Anderson Cancer Center. ....

United States , Ionis Incyte , Prithviraj Bose , University Of Texas Md Anderson Cancer Center , Bristol Myers Squibb , American Society Of Hematology , Manderson Cancer , Annual Meeting , Cogent Biosciences , Blood Disorder , Bone Marrow , Central Nervous System , Mast Cell , Nervous System ,

Mastocytosis - Types, Causes, Symptoms, Diagnosis, Treatment and Prognosis

Mastocytosis is a rare condition marked by accumulation of mast cells under the skin and various organs in the body. Clinical features depend on site affected and treatment is based on specific symptoms in each person. ....

United States , J Clin Dermatol , Smithas Dutt , World Health Organization , Accessed Aug , Mast Cell Disease , Systemic Mastocytosis , Ystemic Mast Cell Disease , Rare Disorder , Mast Cell Disorder ,