Midostaurin (Rydapt)
Multikinase inhibitor (KIT, FLT3, PDGFR, PKC)
- Response rate
- 60% ORR in AdvSM; 45% major response
- Onset
- Weeks–months
- Route
- Oral 100mg twice daily
- Line
- 1st
- IgM effect
- N/A
Evidence summary
First FDA-approved KIT inhibitor for advanced SM (April 2017). In the landmark phase II trial (NEJM 2016), midostaurin achieved 60% ORR with 45% major responses in 89 evaluable AdvSM patients. Median OS 28.7 months. Active against both wild-type and D816V-mutated KIT. Less selective than avapritinib, with higher rates of GI toxicity.