Blueprint Medicines' AYVAKYT® (avapritinib) Receives European Commission Approval for the Treatment of Adults with Advanced Systemic Mastocytosis
-- AYVAKYT is the first approved therapy in the
-- Advanced Systemic Mastocytosis (AdvSM) is a debilitating disease characterized by damage across multiple organ systems, reduced overall survival and poor quality of life --
-- Initial commercial launch is planned for
"Today we are incredibly proud to bring an innovative new treatment option to individuals who have been impacted by advanced systemic mastocytosis," said
About AYVAKYT® (avapritinib)
AYVAKYT® (avapritinib) is a kinase inhibitor approved by the
It is also approved under the brand name AYVAKIT in Mainland China for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations, and in
AYVAKYT/AYVAKIT is not approved for the treatment of any other indication in the
To learn about ongoing or planned clinical trials, contact
Please click here to see the Summary of Product Characteristics for AYVAKYT.
About Systemic Mastocytosis
Systemic mastocytosis (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 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. Across advanced SM subtypes, the median overall survival is approximately 3.5 years in ASM, approximately two years in SM-AHN and less than six months in MCL.5 In
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. Historically, there had been no approved therapies for the treatment of SM that selectively inhibit D816V mutant KIT.7,8
About Blueprint Medicines
Cautionary Note Regarding Forward-Looking Statements
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1 Blueprint Medicines. AYVAKIT (avapritinib) Prescribing Information. Accessed at https://www.blueprintmedicines.com/wp-content/uploads/uspi/AYVAKIT.pdf.
2 CStone Announces China NMPA New Drug Approval of Precision Therapy AYVAKIT® (avapritinib) for the Treatment of Adults with Unresectable or Metastatic PDGFRA Exon 18 Mutant Gastrointestinal Stromal Tumor.
3 "CStone announced new drug approval of precision therapy AYVAKIT® (avapritinib) in
4 "CStone Announces Acceptance of New Drug Application in
5 Sperr WR, Kundi M, Alvarez-Twose I, et al. International prognostic scoring system for mastocytosis (IPSM): a retrospective cohort study. Lancet Haematol. 2019;6(12):e638-e649.
6 Estimated SM prevalence and patient subtypes based on internal claims analysis and epidemiology reported in Orphanet (orpha.net) and Cohen SS, Skovbo S, Vestergaard H, et al. Epidemiology of systemic mastocytosis in
7 Jennings SV, Slee VM, Zack RM, et al. Patient perceptions in mast cell disorders. Immunol Allergy
8 Mesa, RA, Sullivan, EM, Dubinski, D, et al. Patient reported outcomes among systemic mastocytosis (SM) patients in routine clinical practice: results from the
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