Blueprint Medicines Announces EMA Validation of Type II Variation Marketing Authorization Application for AYVAKYT® (avapritinib) for the Treatment of Advanced Systemic Mastocytosis
"AYVAKYT represents a promising approach to address the needs of patients with advanced systemic mastocytosis, a rare, life-threatening disease characterized by severe organ damage," said
AYVAKYT, a potent and selective inhibitor of D816V mutant KIT, is being developed to treat advanced and non-advanced forms of SM. Last month,
SM is a rare disease driven by the KIT D816V mutation. Uncontrolled proliferation and activation of mast cells result in chronic, severe and often unpredictable symptoms for patients across the spectrum of SM. The vast majority of those affected have non-advanced (indolent or smoldering) SM, with debilitating symptoms that lead to a profound, negative impact on quality of life. A minority of patients have advanced SM, which encompasses a group of high-risk SM subtypes including aggressive SM, SM with an associated hematological neoplasm and mast cell leukemia. In addition to mast cell activation symptoms, advanced SM is associated with organ damage due to mast cell infiltration and poor survival.
Debilitating symptoms, including anaphylaxis, maculopapular rash, pruritis, diarrhea, brain fog, fatigue and bone pain, often persist across all forms of SM despite treatment with a number of symptomatic therapies. Patients often live in fear of severe, unexpected symptoms, have limited ability to work or perform daily activities, and isolate themselves to protect against unpredictable triggers. Currently, there are no approved therapies for the treatment of SM that selectively inhibit D816V mutant KIT.
About AYVAKYT (avapritinib)
AYVAKYT (avapritinib) is a kinase inhibitor approved by the
AYVAKIT/AYVAKYT is not approved for the treatment of any other indication, including SM, in the
About Blueprint Medicines
Blueprint Medicines is a global precision therapy company that invents life-changing medicines for people with cancer and hematologic disorders. Applying an approach that is both precise and agile, we create therapies that selectively target genetic drivers, with the goal of staying one step ahead across stages of disease. Since 2011, we have leveraged our research platform, including expertise in molecular targeting and world-class drug design capabilities, to rapidly and reproducibly translate science into a broad pipeline of precision therapies. Today, we are delivering our approved medicines to patients in the United States and Europe, and we are globally advancing multiple programs for genomically defined cancers, systemic mastocytosis, and cancer immunotherapy. For more information, visit www.BlueprintMedicines.com and follow us on Twitter (@BlueprintMeds) and LinkedIn.
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding the potential benefits of AYVAKIT/AYVAKYT in treating patients with SM; plans, timelines and expectations for interactions with the EMA; plans, timelines and expectations for the commercialization of AYVAKYT for the treatment of advanced SM, if approved by the
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