Eliglustat reduces the formation of a certain protein in the body in people with type 1 Gaucher disease.

Gaucher disease is a genetic condition in which the body lacks the enzyme needed to break down certain fatty materials (lipids). Lipids can build up in the body, causing symptoms such as easy bruising or bleeding, weakness, anemia, bone or joint pain, enlarged liver or spleen, or weakened bones that are easily fractured.

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

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

Eliglustat may also be used for purposes not listed in this medication guide.

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.

Your doctor will perform a genotype blood test to make sure eliglustat is the right treatment for you.

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

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

Eliglustat is usually taken 1 or 2 times per day, based 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.

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 twice a day
IM: 84 mg orally twice a day
PM: 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 hepatic impairment taking a weak CYP450 2D6 inhibitor
-EM with mild hepatic impairment taking a strong, moderate, or weak CYP450 3A inhibitor

Comments:
-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 an FDA-cleared test.

Sometimes it is not safe to use certain medications at the same time. Some drugs can raise or lower your blood levels of eliglustat, which may cause side effects or make eliglustat less effective. Eliglustat can also affect blood levels of certain other drugs, making them less effective or increasing side effects.

Your doctor may need to change your treatment plan if you use 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 whether 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 use by anyone younger than 18 years old.

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

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

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

Keep this medication in the container it came in, tightly closed, and out of reach of children. Store it at room temperature and away from excess heat and moisture (not in the bathroom).

It is important to keep all medication out of sight and reach of children as many containers (such as weekly pill minders and those for eye drops, creams, patches, and inhalers) are not child-resistant and young children can open them easily. To protect young children from poisoning, always lock safety caps and immediately place the medication in a safe location – one that is up and away and out of their sight and reach. http://www.upandaway.org

Unneeded medications should be disposed of in special ways to ensure that pets, children, and other people cannot consume them. However, you should not flush this medication down the toilet. Instead, the best way to dispose of your medication is through a medicine take-back program. Talk to your pharmacist or contact your local garbage/recycling department to learn about take-back programs in your community. See the FDA's Safe Disposal of Medicines website (http://goo.gl/c4Rm4p) for more information if you do not have access to a take-back program.

Do not eat grapefruit or drink grapefruit juice while taking this medication.

Grapefruit may interact with eliglustat and lead to unwanted side effects. Avoid the use of grapefruit products.

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

Eliglustat may cause serious side effects. Call your doctor at once if you have:

• sudden dizziness (like you might pass out); or

• fast or pounding heartbeats, 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 occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Eliglustat can cause a serious heart problem. Your risk may be higher if you also use 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 drugs should not be used at the same time. Tell your doctor about all your current medicines and any 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.

Keep all appointments with your doctor and the laboratory. Your doctor may order certain lab tests to check your body's response to eliglustat.

Do not let anyone else take your medication. Ask your pharmacist any questions you have about refilling your prescription.

It is important for you to keep a written list of all of the prescription and nonprescription (over-the-counter) medicines you are taking, as well as any products such as vitamins, minerals, or other dietary supplements. You should bring this list with you each time you visit a doctor or if you are admitted to a hospital. It is also important information to carry with you in case of emergencies.