What Is Arimoclomol and How Does It Work?
Arimoclomol is a prescription medication indicated for the treatment of neurological manifestations of Niemann-Pick disease type C in combination with miglustat in adult and pediatric patients aged 2 years and older.
- Arimoclomol is available under the following different brand names: Miplyffa.
What Are Side Effects Associated with Using Arimoclomol?
Common side effects of Arimoclomol include:
- upper respiratory tract infection
- diarrhea
- decreased weight
- Serious side effects of Arimoclomol include:
- allergic reactions such as swelling and itching
Rare side effects of Arimoclomol include:
- none
Seek medical care or call 911 at once if you have the following serious side effects:
- Severe headache, confusion, slurred speech, arm or leg weakness, trouble walking, coordination loss, unsteady, very stiff muscles, high fever, profuse sweating, or tremors.
- Serious eye symptoms such as sudden vision loss, blurred vision, tunnel vision, eye pain or swelling, or seeing halos around lights.
- Serious heart symptoms include fast, irregular, or pounding heartbeats; fluttering in the chest; shortness of breath; sudden dizziness, lightheadedness, or passing out.
This is not a complete list of side effects and other serious side effects or health problems that may occur because of the use of this drug. Call your doctor for medical advice about serious side effects or adverse reactions. You may report side effects or health problems to FDA at 1-800-FDA-1088.
What Are the Dosages of Arimoclomol?
Adult and pediatric dosage
Capsule
- 47 mg
- 62 mg
- 93 mg
- 124 mg
Niemann-pick disease type C
Adult dosage
- Use actual body weight to calculate the dose
- Weighing more than 30 to 55 kg: 93 mg orally three times a day
- Weighing more than 55 kg: 124 mg orally three times a day
Pediatric dosage
- Children below 2 years: Safety and efficacy not established
- Children aged 2 years and older
- Use actual body weight to calculate the dose
- Weighing between 8 kg and 15 kg: 47 mg orally three times a day
- Weighing more than 15-30 kg: 62 mg orally three times a day
- Weighing more than 30-55 kg: 93 mg orally three times a day
- Weighing more than 55 kg: 124 mg orally three times a day
Dosage Considerations – Should be Given as Follows:
- See "Dosages"
What Other Drugs Interact with Arimoclomol?
If your medical doctor is using this medicine to treat your pain, your doctor or pharmacist may already be aware of any possible drug interactions and may be monitoring you for them. Do not start, stop, or change the dosage of any medicine before checking with your doctor, health care provider, or pharmacist first.
Arimoclomol has severe interactions with no other drugs
Arimoclomol has serious interactions with no other drugs
Arimoclomol has moderate interactions with no other drugs
Arimoclomol has minor interactions with no other drugs
This information does not contain all possible interactions or adverse effects. Visit the RxList Drug Interaction Checker for any drug interactions. Therefore, before using this product, tell your doctor or pharmacist about all the products you use. Keep a list of all your medications with you and share this information with your doctor and pharmacist. Check with your healthcare professional or doctor for additional medical advice, health questions, or concerns.
What Are Warnings and Precautions for Arimoclomol?
Contraindications
None
Effects of drug abuse
None
Short-Term Effects
See “What Are Side Effects Associated with Using Arimoclomol?”
Long-Term Effects
See “What Are Side Effects Associated with Using Arimoclomol?”
Cautions
Hypersensitivity
Hypersensitivity reactions (e.g., urticaria, angioedema) reported during clinical trials within the first 2 months of treatment
Discontinue if severe hypersensitivity reactions occur
If a mild or moderate hypersensitivity reaction occurs, stop arimoclomol and treat promptly; monitor until signs and symptoms resolve
Embryofetal toxicity
May cause embryofetal harm when administered during pregnancy
Advise pregnant women of potential fetal risk
Increased creatinine
Mean increases in serum creatinine (10-20%) occurred compared to baseline; increased creatinine resolved after therapy discontinued
Occurred mostly in the first month of treatment and was not associated with changes in glomerular function
Increases may be due to inhibition of renal tubular secretion transporters
Use other measures not based on creatinine to assess renal function [e.g., Blood urea nitrogen (BUN), cystatin C, glomerular filtration rate (GFR)]
Drug interaction overview
Arimoclomol is an OCT2 inhibitor
OCT2 substrates
Monitor and consider modifying the OCT2 substrate dose
Arimoclomol may increase systemic exposure to OCT2 substrates
Pregnancy and Lactation
Based on findings from animal reproduction studies, arimoclomol may cause embryofetal harm when administered during pregnancy
Advise pregnant women of potential fetal risk
Contraception
Consider pregnancy planning and prevention for females of reproductive potential
Infertility
Based on findings from animal studies, arimoclomol may impair fertility in females and males of reproductive potential
In a rat fertility study, oral administration of arimoclomol resulted in decreased men and women fertility at 9-fold and increased pre-implantation loss at 5-fold the human exposure, based on AUC at MRHD
Unknown if these effects are reversible
Lactation
Data are unavailable regarding the presence of arimoclomol in human or animal milk, its effects on breastfed infants, or milk production
Consider the developmental and health benefits of breastfeeding along with the mother’s clinical need for arimoclomol and potential adverse effects on breastfed infants from the drug or underlying maternal condition
What Other Drugs Interact with Arimoclomol?
If your medical doctor is using this medicine to treat your pain, your doctor or pharmacist may already be aware of any possible drug interactions and may be monitoring you for them. Do not start, stop, or change the dosage of any medicine before checking with your doctor, health care provider, or pharmacist first.
- Arimoclomol has severe interactions with no other drugs
- Arimoclomol has serious interactions with no other drugs
- Arimoclomol has moderate interactions with no other drugs
- Arimoclomol has minor interactions with no other drugs
This information does not contain all possible interactions or adverse effects. Visit the RxList Drug Interaction Checker for any drug interactions. Therefore, before using this product, tell your doctor or pharmacist about all the products you use. Keep a list of all your medications with you and share this information with your doctor and pharmacist. Check with your healthcare professional or doctor for additional medical advice, health questions, or concerns.
What Are Warnings and Precautions for Arimoclomol?
Contraindications
- None
Effects of drug abuse
- None
Short-Term Effects
- See “What Are Side Effects Associated with Using Arimoclomol?”
Long-Term Effects
- See “What Are Side Effects Associated with Using Arimoclomol?”
Cautions
- Hypersensitivity
- Hypersensitivity reactions (e.g., urticaria, angioedema) reported during clinical trials within the first 2 months of treatment
- Discontinue if severe hypersensitivity reactions occur
- If a mild or moderate hypersensitivity reaction occurs, stop arimoclomol and treat promptly; monitor until signs and symptoms resolve
- Embryofetal toxicity
- May cause embryofetal harm when administered during pregnancy
- Advise pregnant women of potential fetal risk
- Increased creatinine
- Mean increases in serum creatinine (10-20%) occurred compared to baseline; increased creatinine resolved after therapy discontinued
- Occurred mostly in the first month of treatment and was not associated with changes in glomerular function
- Increases may be due to inhibition of renal tubular secretion transporters
- Use other measures not based on creatinine to assess renal function [e.g., Blood urea nitrogen (BUN), cystatin C, glomerular filtration rate (GFR)]
- Drug interaction overview
- Arimoclomol is an OCT2 inhibitor
- OCT2 substrates
- Monitor and consider modifying the OCT2 substrate dose
- Arimoclomol may increase systemic exposure to OCT2 substrates
Pregnancy and Lactation
- Based on findings from animal reproduction studies, arimoclomol may cause embryofetal harm when administered during pregnancy
- Advise pregnant women of potential fetal risk
- Contraception
- Consider pregnancy planning and prevention for females of reproductive potential
- Infertility
- Based on findings from animal studies, arimoclomol may impair fertility in females and males of reproductive potential
- In a rat fertility study, oral administration of arimoclomol resulted in decreased men and women fertility at 9-fold and increased pre-implantation loss at 5-fold the human exposure, based on AUC at MRHD
- Unknown if these effects are reversible
- Lactation
- Data are unavailable regarding the presence of arimoclomol in human or animal milk, its effects on breastfed infants, or milk production
- Consider the developmental and health benefits of breastfeeding along with the mother’s clinical need for arimoclomol and potential adverse effects on breastfed infants from the drug or underlying maternal condition
