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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Neha is a Senior Editor at PharmaShots. She is passionate and very enthusiastic about recent updates and developments in the life sciences and pharma industry. She covers Biopharma, MedTech, and Digital health segments along with different reports at PharmaShots. She can be contacted at connect@pharmashots.com.
