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

  • Dec 15, 2023
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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.

Systemic mastocytosis (SM) is a rare hematologic disorder that can lead to a range of debilitating symptoms with a significant impact on patients’ quality of life. The majority of patients living with SM have ISM, and there are approximately 40,000 people living with ISM in the European Union. AYVAKYT was designed to potently and selectively target KIT D816V, the primary underlying driver of the disease.

Today’s approval represents an important step toward delivering a new global standard of care for patients with ISM and builds on years of collaboration with the SM community. For the first time in Europe ISM patients have an approved therapy, marking a new era in the treatment of this disease. AYVAKYT is the first approved medicine for both ISM and advanced SM, and our team is committed to bringing this transformative therapy to patients across the spectrum of disease.”

Georg Pirmin Meyer, M.D., Senior Vice President, International at Blueprint Medicines

“Indolent systemic mastocytosis can be characterized by significant symptom burden across multiple organ systems, which can profoundly impact patients’ ability to perform activities of daily living in a relevant proportion of patients,” said Jens Panse, M.D., Deputy Director of the Department of Hematology/Oncology of the University Hospital RWTH Aachen. “AYVAKYT represents an important treatment breakthrough as the first medicine approved for patients living with ISM, and the only therapy designed to selectively target the primary genetic driver of the disease. In the PIONEER trial, AYVAKYT showed statistically significant and durable clinical benefits across all measured ISM symptoms with a well-tolerated safety profile. Based on these practice-changing data, AYVAKYT has the potential to advance treatment for a broad range of patients living with ISM.”

The approval follows the positive opinion by the Committee for Medicinal Products for Human Use (CHMP), and this EC decision is based on data from the double-blind, placebo-controlled PIONEER trial – the largest study ever conducted in ISM. AYVAKYT showed clinically meaningful improvements versus placebo in the primary and all key secondary endpoints, including overall symptoms and measures of mast cell burden. AYVAKYT was well-tolerated with a favorable safety profile, and most adverse events (AEs) were reported as mild (Grade 1). The most common AEs were flushing, edema, increased blood alkaline phosphate and insomnia.

“Many people living with indolent systemic mastocytosis face unpredictable and severe symptoms, which significantly impair their ability to work or spend quality time with their family, friends and communities,” said Patrizia Marcis, President of the Associazione Italiana Mastocitosi (ASIMAS) ODV. “Today’s approval offers a new sense of hope to the ISM community, and we are proud to collaborate with clinical researchers, patients and companies like Blueprint Medicines to advance care for all those living with the disease.”

In Europe, Blueprint Medicines plans to initiate its first commercial launch in Germany, followed by additional markets based on local healthcare technology assessment and reimbursement process timelines.


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