Blueprint Medicines' AYVAKYT® (avapritinib) Receives Positive CHMP Opinion for the Treatment of Adults with Advanced Systemic Mastocytosis
"The positive opinion from the CHMP is a result of nearly a decade of collaboration with the systemic mastocytosis community and a dedication to bring a transformative therapy to treat and manage this rare, life-threatening disease," said
The CHMP opinion will now be reviewed by the
The CHMP based its opinion on results from the Phase 1 EXPLORER trial and Phase 2 PATHFINDER trial, in which AYVAKYT showed durable clinical efficacy in advanced SM patients across disease subtypes and a generally well-tolerated safety profile. These two studies, which enrolled approximately 150 patients with up to four years of follow-up and comprise the largest ever reported dataset in advanced systemic mastocytosis, were recently published in Nature Medicine.
About AYVAKYT (avapritinib)
AYVAKYT (avapritinib) is a kinase inhibitor approved by the
AYVAKYT/AYVAKIT is not approved for the treatment of any other indication in the Europe, U.S., or Greater China, or for any indication in any other jurisdiction by any other health authority.
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. 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.6,7
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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 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
6 Jennings SV, Slee VM, Zack RM, et al. Patient perceptions in mast cell disorders. Immunol Allergy
7 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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