Blueprint Medicines Published (PIONEER) Trial Results of Ayvakit (avapritinib) for the Treatment of Indolent Systemic Mastocytosis in the NEJM
Shots:
- The (PIONEER) trial evaluating Ayvakit (25mg, qd) + BSC vs PBO + BSC in 212 patients showed rapid, durable & reductions in all measures of pathological mast cell burden, incl. serum tryptase, KIT D816V variant allele fraction and bone marrow mast cells
- Clinical benefits in overall symptoms @24wks. with improvements deepening through 48wks. along with improvements on the most sev. symptom & across all individual symptoms measured, improvements in multiple exploratory EPs of QoL, incl. disease-specific & general health status measures were also reported
- The therapy showed a favorable safety profile, the SAE rate (5.0% vs 11.3%). Ayvakit was approved in the US for adults with ISM
Ref: PRNewswire | Image: Blueprint Medicines
Related News:- Blueprint Medicines’ Ayvakit (avapritinib) Receives the US FDA’s Approval as the First Treatment for Indolent Systemic Mastocytosis
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