Arimoclomol
Arimoclomol is an investigational pharmaceutical compound being developed for the treatment of Niemann-Pick disease type C (NPC), a rare genetic lysosomal storage disorder that causes progressive neurological deterioration. Classified under ATC group N07 (other nervous system drugs), it represents a therapeutic approach for patients suffering from this debilitating condition.
Arimoclomol is an investigational drug for Niemann-Pick disease type C, a rare neurological disorder, though its exact mechanism of action remains unknown.
Key facts at a glance
- ATC
- N07XX17
- RxNorm
- 2694283
- Generic
- Arimoclomol
- Class
- atc-n07
- Brands
- Miplyffa
- Availability
- rx
- Lactation
- unknown
- Routes
- oral
- Forms
- capsule
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Dosage and administration
- Usual dose
- 500mg twice daily
- Maximum
- consult physician
- Frequency
- every 12 hours
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 250mg twice daily
- Maximum
- consult physician
- Frequency
- every 12 hours
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 400mg daily
- Maximum
- consult physician
- Frequency
- once daily
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
Overdose information
Special Populations
8 groupspregnancy
first trimesteravoid if possible
avoid use during pregnancy unless benefits outweigh risks
regular fetal monitoring
Use of this medication in this population requires careful consideration of risks and benefits.
pregnancy
second third trimesteruse with caution
use only if clearly needed and benefits outweigh potential risks
regular fetal monitoring and ultrasound assessments
Use with caution and monitor for adverse effects.
lactation
breastfeedingnot recommended
discontinue breastfeeding or discontinue drug, considering importance of drug to mother
monitor infant for adverse effects
pediatric_age
pediatric 2 to 11 yearsweight-based dosing
approved for use in patients 2 years and older; follow pediatric dosing guidelines
regular growth and developmental monitoring
Use with caution and monitor for adverse effects.
pediatric_age
adolescent 12 to 18 yearsstandard adult dosing
approved for use; follow standard dosing regimen
regular safety monitoring
geriatric
elderly 65 years and olderstart at lower end of dosing range
use with caution due to limited data in elderly population
monitor renal and hepatic function
Use with caution and monitor for adverse effects.
renal_impairment
mild moderate renal impairmentno initial dose adjustment needed
monitor renal function during treatment
monitor renal function periodically
Use with caution and monitor for adverse effects.
hepatic_impairment
mild moderate hepatic impairmentno initial dose adjustment needed
monitor liver function during treatment
monitor hepatic function periodically
Use with caution and monitor for adverse effects.
Statistics and clinical data
FDA Approval Year
FDA approved Arimoclomol (Miplyffa) for Niemann-Pick disease type C
Drug class
Mechanism of action classification
Indication
Approved therapeutic indication in combination with miglustat
Clinical trial phase
Development stage reached for NPC treatment
Arimoclomol is being investigated for the treatment of Niemann-Pick disease type C (NPC), a rare autosomal recessive genetic disorder characterized by abnormal accumulation of cholesterol and lipids in cells, leading to progressive neurological decline including movement disorders, cognitive impairment, and seizures.
The precise mechanism(s) by which arimoclomol exerts its clinical effects in NPC patients remain unknown. Preclinical studies suggest it may work through heat shock protein induction and support of cellular protein folding mechanisms, potentially helping cells clear accumulated lipids more effectively.
Arimoclomol is an investigational drug and has not received regulatory approval. It is being studied in clinical trials to establish its safety, efficacy, and optimal dosing. Patients should only use this medication under supervision in clinical trial settings until more data becomes available.
Contraindications
Conditions and patient states where this medication should be avoided or used with caution.
Do not use (1)
Known allergy to this medication or its components
Use caution (1)
Consult healthcare provider for benefits vs risks assessment
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy