DrugInvestigational line

Bortezomib

Proteasome inhibitor (NF-κB pathway)

Response rate
Single case report
Onset
Unknown
Route
SC or IV
Line
Investigational
IgM effect
Unknown
Evidence level
default

Evidence summary

Novel approach targeting the NF-κB pathway. Bai et al. 2025 reported use in a patient without detectable serum IL-1β who was negative for monogenic autoinflammatory variants and MYD88 L265P. Very preliminary.

Approved indications

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

Multiple MyelomaMantle Cell Lymphoma

Drug identifiers

DrugBankDB00188
ATC CodeL01XG01
Open TargetsCHEMBL325041

Molecular targets

MoleculeRoleExpressionEvidence
NF-κBMaster transcription factorPersistently activestrong

Sources (1)

DetailsBai H et al. (2025) Bortezomib in Schnitzler syndrome without detectable IL-1βDOI