Blueprint Medicines Submits Supplemental New Drug Application to FDA for AYVAKIT® (avapritinib) for the Treatment of Indolent Systemic Mastocytosis
-- Company to present registrational PIONEER trial results at 2023 AAAAI Annual Meeting --
The submission includes data from the registrational PIONEER trial, the largest randomized, placebo-controlled clinical study ever conducted in indolent SM. The FDA has granted breakthrough therapy designation to AYVAKIT for the treatment of moderate to severe indolent SM.
"This regulatory submission for AYVAKIT marks an important milestone to address the medical needs of a broad population of patients with systemic mastocytosis, characterized by debilitating symptoms and lifelong disease burden across multiple organs," said
About AYVAKIT (avapritinib)
AYVAKIT (avapritinib) is a kinase inhibitor approved by the FDA for the treatment of adults with Advanced SM, including aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN) and mast cell leukemia (MCL), and adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. For more information, visit AYVAKIT.com. This medicine is approved in Europe (AYVAKYT®) for the treatment of adults with ASM, SM-AHN or MCL, after at least one systemic therapy, and adults with unresectable or metastatic GIST harboring the PDGFRA D842V mutation. Please click here to see the full U.S. Prescribing Information for AYVAKIT, and click here to see the European Summary of Product Characteristics for AYVAKYT. AYVAKIT/AYVAKYT is not approved for the treatment of any other indication in the U.S. or Europe.
To learn about ongoing or planned clinical trials, contact Blueprint Medicines at firstname.lastname@example.org or 1-888-BLU-PRNT (1-888-258-7768). Additional information is available at blueprintclinicaltrials.com or clinicaltrials.gov.
About Systemic Mastocytosis
Systemic mastocytosis (SM) is a rare disease primarily driven by the KIT D816V mutation. Uncontrolled proliferation and activation of mast cells result in chronic, severe and often unpredictable symptoms across multiple organ systems. The vast majority of those affected have non-advanced (indolent or smoldering) SM. A broad range of symptoms, including anaphylaxis, maculopapular rash, pruritis, diarrhea, brain fog, fatigue and bone pain, frequently persist in patients with non-advanced SM despite treatment with multiple symptom-directed therapies. This burden of disease can lead to a profound, negative impact on quality of life. 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 non-advanced SM.
A minority of patients have advanced SM, which encompasses a group of high-risk SM subtypes including ASM, SM-AHN and MCL. In addition to mast cell activation symptoms, advanced SM is associated with organ damage due to mast cell infiltration and poor survival.
About Blueprint Medicines
Blueprint Medicines is a global precision therapy company that invents life-changing therapies for people with cancer and blood disorders. Applying an approach that is both precise and agile, we create medicines 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 our scientific innovation into a broad pipeline of important approved and investigational precision therapies aimed at addressing difficult-to-treat cancers and blood disorders. Today, we are delivering our approved medicines to patients in the United States, Europe, and in other geographies ourselves or through our partners. In addition, we are globally advancing multiple programs for systemic mastocytosis, lung cancer, breast cancer, and other genomically defined cancers, 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 submission of a supplemental new drug application to the FDA for AYVAKIT for the treatment of indolent SM; plans and timing for presenting detailed data from the PIONEER trial of AYVAKIT in patients with non-advanced SM, and, expectations regarding the potential benefits of AYVAKIT in treating patients with non-advanced SM; statements regarding plans, strategies, timelines and expectations for interactions with the FDA and other regulatory authorities; statements regarding plans and expectations for
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