Drug2nd line

Venetoclax

BCL2 inhibitor

Response rate
~70% MRR (relapsed/refractory)
Onset
Weeks-months
Route
Oral (dose escalation to 400mg daily)
Line
2nd
IgM effect
Significant reduction
Evidence level
amber

Evidence summary

BCL2 inhibitor with activity in relapsed/refractory WM, including after BTK inhibitor failure. Being studied in combination with ibrutinib (fixed-duration) and pirtobrutinib. TP53 mutations may predict inferior outcomes. Requires dose escalation due to tumour lysis syndrome risk.

Approved indications

Conditions for which Venetoclax has regulatory approval (not specific to rare diseases covered here):

CLLAMLMantle Cell Lymphoma

Drug identifiers

DrugBankDB11581 ↗
ATC CodeL01XX52