The P-II trial evaluating Ayvakit (25mg, qd) + BSC vs PBO + BSC in 251 patients at 49 sites in 13 countries showed an improvement on 1EPs & 2EPs i.e., a rapid, durable & reduction on all pathological mast cell burden measures @24wks.
≥50% reduction in serum tryptase/KIT D816V variant allele fraction/bone marrow mast cell aggregates (53.9% vs 0%)/ (67.8% vs 6.3%)/ (52.8% vs 22.8%), ≥30% & ≥50% reduction in TSS (45.4% @24wks. & 60.7% @48wks. vs 29.6% @24wks.) & (24.8% & 39.3% vs 9.9%)
Improvement in mean change in most sev. symptom score, mean percent change in MC-QoL total score (-34.3% vs -17.9%), was well-tolerated with a favorable safety profile, SAEs (11.3% vs 5.0%). The company submitted Ayvakit’ sNDA to the US FDA & type II variation MAA to the EMA. The US FDA has granted priority review to Ayvakit’ sNDA with a PDUFA of May 2023
Ref: PRNewswire | Image: Blueprint Medicines
Related News:- Blueprint Medicines Receives the US FDA’s Acceptance of sNDA for Ayvakit (avapritinib) to Treat Indolent Systemic Mastocytosis
