Blueprint Medicines Reports (PIONEER) Trial Results of Ayvakit (avapritinib) for the Treatment of Non-Advanced Systemic Mastocytosis
Shots:
- Part 2 of the (PIONEER) trial evaluates Ayvakit (25mg, qd) + best available care vs PBO + best available care in 212 patients with non-advanced SM for 24wk.
- The trial met its 1EPs & 2EPs i.e., 15.6 points vs 9.2 points reduction in TSS @24wks., improvements across all measures of mast cell burden, ≥50% reduction of serum tryptase (53.9% vs 0%), was well-tolerated & had a favorable safety profile, 96.5% vs 93.0% has completed 24wks. of therapy, 0.7% vs 0% discontinued due to TRAEs, rate of AEs (90.8% vs 93.0)
- The company plans to submit an sNDA of Ayvakit to the US FDA in non-advanced SM in Q4’22 with an expected submission of a type II variation MAA to the EMA in 2023. The (PIONEER) trial results will be presented at an upcoming medical meeting
Ref: Blueprint Medicines | Image: Blueprint Medicines
Click here to read the full press release
Tags
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.