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arimoclomol + miglustat
- Development stage
- Phase 3
- Lead developer
- Johnson & Johnson
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
Arimoclomol + miglustat is an orally administered combination regimen indicated for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients aged 2 years and older. Arimoclomol (marketed as Miplyffa) is a small-molecule heat shock protein co-inducer that crosses the blood-brain barrier and enhances the cellular stress response by stabilizing active heat shock factor 1 (HSF1). This stabilization leads to the upregulation of molecular chaperones like HSP70 and transcription factors TFEB and TFE3, which improve lysosomal function and clear lipid aggregates. Miglustat (marketed as Zavesca) is a small-molecule inhibitor of ceramide glucosyltransferase (glucosylceramide synthase) that acts as a substrate reduction therapy to decrease the biosynthesis and accumulation of glycosphingolipids. Together, the combination slows or halts the progressive neurodegeneration characteristic of NPC.
- Brand names
- Brazaves
- Other names
- Miplyffa + ZavescaMiplyffa and ZavescaMiplyffa/ZavescaN-butyldeoxynojirimycinNB-DNJarimoclomol and miglustatarimoclomol/miglustatbutyldeoxynojirimycin
02
Targets
UGCG (UDP-glucose ceramide glucosyltransferase)HSPA1A (HSP70-1A)HSF1 (Heat shock factor protein 1)TFE3 (Transcription factor E3)TFEB (Transcription factor EB)
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