Arimoclomol
NA
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Drug Status
Government Approvals
US(FDA)
WHO Essential Medicine
NO
Known Teratogen
No
Pharmaceutical Class
NA
Controlled Drug Substance
No
Arimoclomol
Summary
Arimoclomol is used to manage symptoms of Niemann-Pick disease type C, a genetic disorder affecting the body's ability to metabolize lipids.
Arimoclomol is a heat shock protein activator. It helps manage neurological symptoms by assisting in protein folding and cellular stress responses, although its exact mechanism is not fully understood.
The dose of Arimoclomol varies based on body weight, taken three times a day. For example, for weights over 55 kg, the dose is 124 mg three times a day. It can be taken with or without food.
Common side effects include upper respiratory tract infections, diarrhea, weight loss, and headaches. Serious adverse effects can include hypersensitivity reactions like urticaria and angioedema.
Arimoclomol may harm a fetus and should be avoided by pregnant women. Women of reproductive potential should use effective contraception. It can interact with drugs that are substrates of the OCT2 transporter. It may also cause hypersensitivity reactions.
Indications and Purpose
How does Arimoclomol work?
Arimoclomol is a heat shock protein activator, which helps manage neurological symptoms in Niemann-Pick disease type C. Its exact mechanism of action is not fully understood, but it is believed to assist in protein folding and cellular stress responses.
Is Arimoclomol effective?
Arimoclomol's effectiveness was evaluated in a 12-month trial involving patients with Niemann-Pick disease type C. The trial showed that Arimoclomol, in combination with miglustat, helped manage neurological symptoms. Patients experienced less progression in disease severity compared to those on placebo.
What is Arimoclomol?
Arimoclomol is used to treat neurological symptoms in patients with Niemann-Pick disease type C. It belongs to a class of medications called heat shock protein activators, though its exact mechanism is not fully understood. It helps manage symptoms but does not cure the disease.
Directions for Use
For how long do I take Arimoclomol?
Arimoclomol is used to manage symptoms of Niemann-Pick disease type C and is typically taken long-term as it controls but does not cure the disease. Continue taking it as prescribed by your doctor.
How do I take Arimoclomol?
Arimoclomol can be taken with or without food, three times a day. There are no specific food restrictions, but it's important to take it at the same times each day. If you have trouble swallowing capsules, you can mix the contents with water, apple juice, or soft food.
How should I store Arimoclomol?
Store Arimoclomol in its original container at room temperature, between 68°F to 77°F (20°C to 25°C). Keep it away from light, excess heat, and moisture, and out of reach of children.
What is the usual dose of Arimoclomol?
The usual daily dose of Arimoclomol varies based on body weight. For those weighing 8 kg to 15 kg, the dose is 47 mg three times a day. For weights over 15 kg to 30 kg, it's 62 mg three times a day. For weights over 30 kg to 55 kg, it's 93 mg three times a day. For those over 55 kg, the dose is 124 mg three times a day. Always follow your doctor's specific instructions.
Warnings and Precautions
Can Arimoclomol be taken safely while breastfeeding?
There is no data on the presence of Arimoclomol in human milk or its effects on a breastfed infant. Consider the benefits of breastfeeding along with the mother's need for Arimoclomol and any potential risks to the infant.
Can Arimoclomol be taken safely while pregnant?
Arimoclomol may cause harm to a fetus, as shown in animal studies. Pregnant women should avoid using it, and women of reproductive potential should use effective contraception. If you become pregnant while taking Arimoclomol, contact your doctor immediately.
Can I take Arimoclomol with other prescription drugs?
Arimoclomol can interact with drugs that are substrates of the OCT2 transporter, potentially increasing their exposure. If you are taking such medications, your doctor may need to adjust the dosage or monitor for side effects.
Who should avoid taking Arimoclomol?
Important warnings for Arimoclomol include the risk of hypersensitivity reactions and potential harm to a fetus if taken during pregnancy. Patients should be monitored for increased creatinine levels. It's crucial to inform your doctor of any allergies or kidney issues before starting treatment.
