Eliglustat
Gaucher Disease
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Drug Status
Government Approvals
US(FDA), UK(BNF)
WHO Essential Medicine
NO
Known Teratogen
No
Pharmaceutical Class
Glucosylceramide Synthase Inhibitor
Controlled Drug Substance
No
Eliglustat
Summary
Eliglustat is used for the long-term treatment of Gaucher disease type 1, a condition where a fatty substance builds up in organs. It helps manage the disease but does not cure it.
Eliglustat works by inhibiting the enzyme glucosylceramide synthase. This enzyme is responsible for producing a fatty substance that accumulates in Gaucher disease type 1. By reducing its production, Eliglustat helps decrease its buildup in organs, alleviating symptoms and improving health outcomes.
Eliglustat is taken orally. For adults, the dose is based on their CYP2D6 metabolizer status. Extensive and intermediate metabolizers typically take 84 mg twice daily while poor metabolizers take 84 mg once daily. For children aged 6 to 18 years weighing 25 kg, dosing is based on weight and metabolizer status. Always follow your doctor's specific dosage instructions.
Common side effects of Eliglustat include fatigue, headache, nausea, diarrhea, and back pain. Serious side effects can include heart palpitations, dizziness or fainting. If you experience any serious side effects, contact your doctor immediately.
Eliglustat is contraindicated in patients with certain CYP2D6 metabolizer statuses when taking specific inhibitors due to the risk of cardiac arrhythmias. It should be avoided in patients with preexisting cardiac conditions such as long QT syndrome and those taking certain antiarrhythmic medications. Patients should avoid grapefruit products and discuss all medications with their doctor to prevent interactions.
Indications and Purpose
How does Eliglustat work?
Eliglustat works by inhibiting an enzyme involved in the production of certain fats in the body. This helps reduce the accumulation of these fats in organs and tissues, which is a problem in Gaucher disease type 1. Think of it like turning down the production line in a factory to prevent excess buildup. By reducing fat buildup, Eliglustat helps manage symptoms like enlarged organs and bone pain, improving quality of life.
Is Eliglustat effective?
Eliglustat is effective for treating Gaucher disease type 1, which is a genetic disorder affecting fat metabolism. Clinical studies show that Eliglustat helps reduce spleen and liver size, improve blood counts, and decrease bone pain in people with this condition. It works by inhibiting an enzyme involved in the production of certain fats. This helps manage symptoms and improve quality of life for those with Gaucher disease type 1.
What is Eliglustat?
Eliglustat is a medication used to treat Gaucher disease type 1, which is a genetic disorder affecting fat metabolism. It belongs to a class of drugs called enzyme inhibitors. Eliglustat works by reducing the production of certain fats in the body, helping to manage symptoms like enlarged organs and bone pain. It's used as a long-term treatment to improve quality of life for those with Gaucher disease type 1.
Directions for Use
For how long do I take Eliglustat?
Eliglustat is usually a long-term medication for managing Gaucher disease type 1, which is a chronic condition. You'll typically take Eliglustat every day as a lifelong treatment unless your doctor suggests otherwise. How long you'll need this medication depends on your body's response, any side effects you experience, and changes in your overall health. Always talk with your doctor before changing or stopping your Eliglustat treatment.
How do I dispose of Eliglustat?
To dispose of Eliglustat, take it to a drug take-back program or collection site at a pharmacy or hospital. If that's not possible, you can throw it in the trash at home. First, remove it from its original container, mix it with something undesirable like used coffee grounds, seal the mixture in a plastic bag, and throw it away. This helps prevent accidental ingestion or harm to the environment.
How do I take Eliglustat?
Take Eliglustat as prescribed by your doctor, usually once or twice daily. It's important to follow your doctor's instructions regarding whether to take it with food or on an empty stomach. Do not crush or chew the capsules. If you miss a dose, take it as soon as you remember unless it's almost time for your next dose. In that case, skip the missed dose and continue your regular schedule. Avoid taking two doses at once.
How long does it take for Eliglustat to start working?
Eliglustat begins working in your body shortly after you take it, but you may not notice all the benefits right away. Improvements in symptoms like reduced spleen and liver size or better blood counts may take several weeks to months. How quickly the medication works can depend on your overall health and how your body responds. Regular check-ups with your doctor are important to monitor your progress and adjust treatment as needed.
How should I store Eliglustat?
Store Eliglustat at room temperature, away from moisture and light. Keep it in its original container, tightly closed, and out of reach of children. Avoid storing it in humid places like bathrooms. Check the expiration date regularly and properly dispose of any unused or expired medication. If you have any questions about storage, consult your pharmacist or healthcare provider.
What is the usual dose of Eliglustat?
The usual starting dose of Eliglustat for adults is typically 84 mg taken twice daily. Your doctor may adjust the dose based on your specific needs and response to the medication. It's important to follow your doctor's dosing instructions carefully. Always consult your doctor for personalized dosing recommendations, especially if you have other health conditions or are taking other medications.
Warnings and Precautions
Can Eliglustat be taken safely while breastfeeding?
Eliglustat is not recommended while breastfeeding due to limited information on its safety. It's unclear if the medication passes into breast milk or affects milk production. If you're taking Eliglustat and want to breastfeed, talk with your doctor about safer medication options. They can help you make an informed decision that ensures the safety of your baby while managing your health condition.
Can Eliglustat be taken safely while pregnant?
Eliglustat is not recommended during pregnancy due to limited evidence on its safety. Animal studies suggest potential risks, but human data is lacking. Uncontrolled Gaucher disease during pregnancy can cause complications for both mother and baby. If you're pregnant or planning to become pregnant, talk with your doctor about the safest way to manage your condition. Your doctor can help create a treatment plan that protects both you and your baby.
Can I take Eliglustat with other prescription drugs?
Eliglustat can interact with certain medications, which may increase the risk of adverse effects or reduce its effectiveness. It's important to inform your doctor about all the medications you are taking, including prescription, over-the-counter, and herbal products. Your doctor can help identify any potential interactions and adjust your treatment plan accordingly to ensure safety and effectiveness.
Does Eliglustat have adverse effects?
Adverse effects are unwanted reactions to a medication. Eliglustat can cause some adverse effects, though most people tolerate it well. Common adverse effects include fatigue and headache. Serious side effects are rare but can include heart problems or liver issues. If you notice any new or worsening symptoms, contact your doctor immediately. They can help determine if the symptoms are related to Eliglustat and advise on the best course of action.
Does Eliglustat have any safety warnings?
Yes, Eliglustat has important safety warnings. It may not be suitable for people with certain medical conditions, such as liver or kidney problems. Not adhering to safety warnings can lead to serious health issues. Always follow your doctor's advice and report any unusual symptoms. If you experience severe side effects, seek medical attention immediately.
Is Eliglustat addictive?
Eliglustat is not considered addictive or habit-forming. It does not cause dependency or withdrawal symptoms when you stop taking it. This medication works by affecting certain enzymes in the body and does not impact brain chemistry in a way that leads to addiction. If you have concerns about medication dependence, discuss them with your doctor.
Is Eliglustat safe for the elderly?
Elderly individuals may be more vulnerable to the side effects of Eliglustat due to age-related changes in metabolism and organ function. While Eliglustat can be used by older adults, careful monitoring is important. Specific risks include dizziness and fatigue, which may be more pronounced in the elderly. Regular check-ups with a healthcare provider can help ensure the medication is used safely and effectively in older patients.
Is it safe to drink alcohol while taking Eliglustat?
It's best to avoid alcohol while taking Eliglustat. Alcohol can interfere with the medication's effectiveness and increase the risk of side effects. Drinking alcohol may also worsen symptoms like dizziness or low blood pressure. If you choose to drink occasionally, limit your alcohol intake and watch for warning signs like nausea or dizziness. Talk with your doctor about alcohol use while taking Eliglustat for personalized advice.
Is it safe to exercise while taking Eliglustat?
You can exercise while taking Eliglustat, but be mindful of how your body responds. This medication can cause dizziness or fatigue, which might affect your exercise capacity. To exercise safely, stay hydrated and avoid strenuous activities if you feel unwell. Listen to your body and rest if you experience dizziness or unusual fatigue. Consult your doctor if you have concerns about exercising while on Eliglustat.
Is it safe to stop Eliglustat?
It's important not to stop taking Eliglustat without consulting your doctor. Stopping suddenly can lead to a return of symptoms or worsening of your condition. Your doctor may suggest gradually reducing your dose or switching to a different medication to keep your condition under control. Always talk with your doctor before making any changes to your medication regimen.
What are the most common side effects of Eliglustat?
Side effects are unwanted reactions to a medication. Common side effects of Eliglustat include fatigue, headache, and nausea. These effects vary from person to person. If you notice new symptoms after starting Eliglustat, they might be temporary or unrelated to the medication. Talk with your doctor before stopping any medication. They can help determine if the side effects are related to Eliglustat and suggest ways to manage them.
Who should avoid taking Eliglustat?
Eliglustat should not be used by people with severe liver impairment or those who are poor metabolizers of certain enzymes. These are absolute contraindications due to severe risks. Relative contraindications include moderate liver impairment, where caution is needed, and the drug may be used only if benefits outweigh risks. Always consult your doctor about any concerns or conditions you have before starting Eliglustat.
