Pharma Focus Asia

Blueprint Medicines Introduce AYVAKYT® First and Only Treatment for Indolent Systemic Mastocytosis

Blueprint Medicines has introduced AYVAKYT® (avapritinib), the first and only therapy for adults experiencing indolent systemic mastocytosis (ISM) with moderate to severe symptoms that have not been effectively managed with current symptomatic treatments.

AYVAKYT stands only approved therapy for individuals with Indolent systemic mastocytosis (ISM) in Europe. It is the premier sanctioned medication for both ISM and advanced SM.

AYVAKYT® (avapritinib), a kinase inhibitor, has gained approval for three treatments including adults with indolent systemic mastocytosis (ISM); adults dealing with aggressive systemic mastocytosis (ASM), systemic mastocytosis associated with a haematological neoplasm (SM-AHN), or mast cell leukaemia (MCL) post at least one systemic therapy; and adults confronting unresectable or metastatic gastrointestinal stromal tumours (GIST) carrying the PDGFRA D842V mutation.

Indolent systemic mastocytosis often presents with a substantial burden of symptoms affecting various organ systems. This array of symptoms can profoundly disrupt the daily lives of a significant portion of patients, hindering their ability to carry out routine activities.

Systemic mastocytosis (SM) is indeed a rare haematologic disorder that can cause various challenging symptoms, significantly affecting patients' daily lives. The most common form of SM is indolent systemic mastocytosis (ISM), which affects a majority of patients, with an estimated 40,000 individuals living with ISM in the European Union alone.

AYVAKYT is developed to address this condition by targeting KIT D816V, which is the primary driver of the disease. This targeted approach aims to manage symptoms and potentially improve the quality of life for those affected by ISM.

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