Drug1st line

Zanubrutinib

Second-generation selective BTK inhibitor

Response rate
~95% ORR; 36% VGPR+CR
Onset
Weeks
Route
Oral 160mg BID or 320mg daily
Line
1st
IgM effect
Significant reduction
Evidence level
green

Evidence summary

Next-generation BTK inhibitor with greater selectivity for BTK. The ASPEN phase III trial demonstrated comparable efficacy to ibrutinib with significantly improved safety: less atrial fibrillation (7.9% vs 23.5%), less hypertension, less diarrhea. Currently recommended as preferred first-line BTK inhibitor by Mayo Clinic guidelines.

Approved indications

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

Waldenström's MacroglobulinemiaCLL/SLLMantle Cell LymphomaMarginal Zone Lymphoma

Drug identifiers

DrugBankDB15035
ATC CodeL01EL03

Molecular targets

MoleculeRoleExpressionEvidence
BTK (Bruton's Tyrosine Kinase)Key signaling kinase downstream of MYD88Constitutively activatedestablished