Drug1st line
Avapritinib (Ayvakit)
Selective type 1 KIT D816V inhibitor
- Response rate
- PIONEER: significant symptom reduction vs placebo; AdvSM: 30% molecular response
- Onset
- Weeks
- Route
- Oral 25mg daily (ISM) / 200mg daily (AdvSM)
- Line
- 1st
- IgM effect
- N/A
- Evidence level
- green
- Trial phase
- Phase 4
Evidence summary
Highly selective inhibitor of KIT D816V with potent activity against the mutant receptor. First and only FDA-approved therapy for ISM (May 2023). In PIONEER trial, avapritinib 25mg daily significantly reduced symptoms (TSS -15.6 vs -9.2, P<0.003) and achieved >=50% tryptase reduction in 54% of patients vs 0% placebo. In AdvSM, achieved molecular responses with KIT D816V becoming undetectable in 30% of patients.
Approved indications
Conditions for which Avapritinib (Ayvakit) has regulatory approval (not specific to rare diseases covered here):
Indolent systemic mastocytosisAdvanced systemic mastocytosisPDGFRA-rearranged GIST
Drug identifiers
| DrugBank | DB15233 ↗ |
| ATC Code | L01EX18 |