Section 12 · Monograph
Arimoclomol
Reviewed Nov 2026FDA label
MiplyffaNPC therapy
- Forms
- Cap: 47, 62, 93, 124mg
- Dose
- Niemann-Pick type C (with miglustat), age 2+: weight-based, three times daily — 8-15kg 47mg; over 15-30kg 62mg; over 30-55kg 93mg; over 55kg 124mg
- MOA
- Mechanism of action not established
Indications
- Neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients 2 years of age and older, in combination with miglustatFDA-labeled
Mechanism of Action
- The mechanism by which arimoclomol exerts its clinical effect in NPC is not known
Full Dosing
Adult / usual
- Weight-based, three times daily: 8-15kg 47mg; over 15-30kg 62mg; over 30-55kg 93mg; over 55kg 124mg
- Dosage is based on actual body weight and used together with miglustat
- Not used together with levacetylleucine (Aqneursa) per payer policies
Contraindications
- None
Drug Interactions
- Used in combination with miglustat as labeled
- Increases serum creatinine without changing true GFR (inhibits renal tubular secretion) — interpret renal function with this in mind
Adverse Effects
- Upper respiratory tract infection
- Diarrhea
- Decreased weight
- Hypersensitivity reactions (urticaria)
- Seizure and tremor reported
Monitoring Parameters
- Use creatinine-independent measures (cystatin C or measured GFR) to assess renal function
- Hypersensitivity reactions
- Weight and neurologic status (e.g., 4-domain NPC Clinical Severity Scale)
Pregnancy & Lactation
- Limited human data; consult the current label and consider embryo-fetal risk
- Unknown whether present in human milk