Shots:

• The full FDA approval is based on data from 2 clinical trials i.e.- P-I EXPLORER & P-II PATHFINDER trial evaluating the efficacy of Ayvakit (200 mg- PO- qd) in 53 adults with advanced SM including ASM- SM-AHN & MCL
• Results: @median follow-up of 11.6 mos.- ORR (57%)- CR/CRh (28%)- PR (28 %) & clinical improvement (15%)- m-DoR (38.3 mos.). Treatment response was evaluated using modified IWG-MRT-ECNM criteria & additional efficacy measures are DoR- resolution- >50% reductions in biomarker response- mast cell burden & serum tryptase
• Ayvakit is not recommended for patients with low platelet counts (<50-000/µL). This approval marks the 1^st targeted therapy that inhibits D816V mutant KIT

  | Ref: Blueprint Medicines | Image: PR Newswire