Eliglustat

Eliglustat reduces the production of a specific protein in the body for people with type 1 Gaucher disease.

Gaucher disease is a genetic disorder where the body does not have the enzyme needed to break down certain fatty substances (lipids). These lipids can accumulate in the body, leading to symptoms like easy bruising or bleeding, weakness, anemia, bone or joint pain, enlarged liver or spleen, or fragile bones that can break easily.

Eliglustat is used to treat mild to moderate type 1 Gaucher disease in adults. Eliglustat is only used if a specific liver enzyme (2D6) in your body processes drugs at a certain speed.

Eliglustat may help improve the health of the liver, spleen, bones, and blood cells in people with type 1 Gaucher disease. However, eliglustat is not a cure for this condition.

Eliglustat may also be used for reasons not mentioned in this medication guide.

Your doctor will do a genotype blood test to ensure eliglustat is the right treatment for you.

Follow all instructions on your prescription label and read all medication guides or instruction sheets. Your doctor may change your dose from time to time. Use the medicine exactly as directed.

If you have switched to eliglustat from another enzyme replacement medicine, wait at least 24 hours after your last dose of the other medicine before starting eliglustat.

Eliglustat is usually taken 1 or 2 times per day, depending on the results of your genotype test. Follow your doctor's dosing instructions very carefully.

Take this medicine with a full glass of water.

Take the medicine at the same time each day, with or without food.

Swallow the capsule whole and do not crush, chew, break, or open it.

Eliglustat can cause a serious heart problem. Your heart function may be checked with an electrocardiograph or ECG (sometimes called an EKG) before you take this medicine.

Store at room temperature away from moisture and heat.

You should not use eliglustat if you have severe liver or kidney disease.

Eliglustat can cause a serious heart problem, especially if you use certain other medicines at the same time. Tell your doctor about all medicines you use.

Sometimes it is not safe to use certain medications together. Some drugs can change the amount of eliglustat in your blood, which may lead to side effects or reduce how well eliglustat works. Eliglustat can also affect the blood levels of some other drugs, making them less effective or causing more side effects.

Your doctor may need to adjust your treatment plan if you take any of the following drugs:

• St. John's wort;
• antifungal medicine;
• tuberculosis medicine;
• seizure medicine;
• heart or blood pressure medications;
• an antidepressant; or
• medicine to treat a mental illness.

You should not use eliglustat if you are allergic to it, or if you have:

• severe liver disease.

Tell your doctor if you have ever had:

• long QT syndrome or other heart rhythm disorder;
• heart disease or a heart attack;
• liver disease; or
• kidney disease.

It is not known if eliglustat will harm an unborn baby. Tell your doctor if you are pregnant or plan to become pregnant.

You should not breast-feed while using this medicine.

Eliglustat is not approved for anyone younger than 18 years old.

Get emergency medical help if you notice signs of an allergic reaction: hives; trouble breathing; swelling of your face, lips, tongue, or throat.

Eliglustat may cause serious side effects. Call your doctor right away if you experience:

• sudden dizziness (feeling like you might faint); or
• fast or pounding heartbeats, or fluttering in your chest.

Common side effects of eliglustat may include:

• diarrhea, nausea, stomach pain;
• headache;
• tiredness;
• back pain; or
• pain in your arms or legs.

This is not a complete list of side effects and others may happen. Call your doctor for medical advice about side effects. You can report side effects to the FDA at 1-800-FDA-1088.

Usual Adult Dose for Gaucher Disease:

Dose recommendations are provided based on CYP450 2D6 Metabolizer Status:Extensive metabolizers (EM); Intermediate metabolizers (IM); Poor metabolizers (PM)EM: 84 mg orally 2 times a dayIM: 84 mg orally 2 times a dayPM: 84 mg orally once a day Dose Reduction: 84 mg orally once a day recommended for:-EM or IM taking a strong or moderate CYP450 2D6 inhibitors-EM taking a strong or moderate CYP450 3A inhibitor-EM with mild liver dysfunction taking a weak CYP450 2D6 inhibitor-EM with mild liver dysfunction taking a strong, moderate, or weak CYP450 3A inhibitorComments: -CYP450 2D6 ultra-rapid metabolizers (URM) may not achieve adequate drug concentrations to achieve a therapeutic effect.-Dose recommendations cannot be provided for patients whose genotype cannot be determined (indeterminate metabolizers).Use: For the long-term treatment of patients with Gaucher disease type 1 who are CYP450 2D6 extensive metabolizers, intermediate metabolizers, or poor metabolizers as detected by a US FDA-cleared test.

Take the medicine as soon as you can, but skip the missed dose if it is close to the time for your next dose. Do not take two doses at the same time.

Get your prescription refilled before you run out of medicine entirely.

Seek emergency medical attention or call the Poison Help line at 1-800-222-1222.

Grapefruit may interact with eliglustat and cause unwanted side effects. Do not use grapefruit products.

Eliglustat can cause a serious heart problem. Your risk may be higher if you also take certain other medicines for infections, asthma, heart problems, high blood pressure, depression, mental illness, cancer, malaria, or HIV.

Many drugs can affect eliglustat, and some should not be used together. Inform your doctor about all the medicines you are currently taking and any new medicine you start or stop using. This includes prescription and over-the-counter medicines, vitamins, and herbal products. Not all possible interactions are listed here.