Eliglustat

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

Call your doctor at once if you have:

• headache with chest pain;
• a light-headed feeling, like you might pass out; or
• pounding heartbeats or fluttering in your chest.

Common side effects may include:

• diarrhea, nausea, stomach pain;
• headache;
• 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.

Serious side effects have been reported with eliglustat. See the “Eliglustat Precautions” section.

Common side effects of eliglustat include fatigue, headache, nausea, diarrhea, back pain, pain in the extremities, and upper abdominal pain.

This is not a complete list of eliglustat side effects. Ask your doctor or pharmacist for more information.

Tell your doctor if you have any side effect that bothers you or that does not go away.

Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.

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

TELL YOUR DOCTOR ABOUT ALL OTHER MEDICINES YOU USE. 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.

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

If you will be taking eliglustat for a long time, it is very important that your doctor check you at regular visits for any problems or unwanted effects that may be caused by eliglustat.

Contact your doctor right away if you have any changes to your heart rhythm. You might feel dizzy or faint, or you might have a fast, pounding, or uneven heartbeat. Make sure your doctor knows if you or anyone in your family has ever had a heart rhythm problem such as QT prolongation.

Do not take other medicines unless they have been discussed with your doctor. This includes prescription or nonprescription (over-the-counter [OTC]) medicines and herbal (eg, St. John's wort) or vitamin supplements.

WARNING/CAUTION: Even though it may be rare, some people may have very bad and sometimes deadly side effects when taking a drug. Tell your doctor or get medical help right away if you have any of the following signs or symptoms that may be related to a very bad side effect:

• Signs of an allergic reaction, like rash; hives; itching; red, swollen, blistered, or peeling skin with or without fever; wheezing; tightness in the chest or throat; trouble breathing or talking; unusual hoarseness; or swelling of the mouth, face, lips, tongue, or throat.
• A fast heartbeat or passing out.
• A heartbeat that does not feel normal.
• Dizziness.

Excipient information presented when available (limited, particularly for generics); consult specific product labeling.

Capsule, Oral:

Cerdelga: 84 mg [contains fd&c blue #2 (indigotine)]

Mild renal impairment: No dosage adjustment necessary.

Moderate to severe renal impairment: Use is not recommended (has not been studied).

End-stage renal disease (ESRD): Use is not recommended (has not been studied).

Store at 20ºC to 25ºC (68ºF to 77ºF); excursions are permitted between 15ºC and 30ºC (59ºF and 86ºF).

Concerns related to adverse effects:

• Arrhythmias: May cause increases in ECG intervals (PR, QTc, and QRS) at substantially elevated eliglustat plasma concentrations.

Disease-related concerns:

• Cardiovascular disease: Use is not recommended in patients with preexisting cardiac disease (CHF, recent acute MI, bradycardia, heart block, ventricular arrhythmia), long QT syndrome, and in combination with Class IA (eg, quinidine, procainamide) and Class III (eg, amiodarone, sotalol) antiarrhythmic medications (has not been studied).

• Hepatic impairment: Not recommended in hepatic impairment or cirrhosis (has not been studied).

• Renal impairment: Not recommended in patients with moderate to severe renal impairment or end-stage renal disease (ESRD); use with caution in patients with mild renal impairment.

• Poor metabolizers: Dosing has not been studied in poor metabolizers (PMs); however, the predicted systemic exposures in these patients are within the range of those observed in clinical studies; monitor these patients for adverse reactions.

Concurrent drug therapy issues:

• Drug-drug interactions: Potentially significant interactions may exist, requiring dose or frequency adjustment, additional monitoring, and/or selection of alternative therapy. Consult drug interactions database for more detailed information.

Other warnings/precautions:

• Registry: A registry has been established and all patients with Gaucher disease, and health care providers who treat Gaucher disease are encouraged to participate. Information on the International Collaborative Gaucher Group (ICGG) Gaucher Registry may be obtained at https://www.registrynxt.com or by calling 1-800-745-4447 (ext.15500).

• Discuss specific use of drug and side effects with patient as it relates to treatment. (HCAHPS: During this hospital stay, were you given any medicine that you had not taken before? Before giving you any new medicine, how often did hospital staff tell you what the medicine was for? How often did hospital staff describe possible side effects in a way you could understand?)

• Patient may experience abdominal pain, diarrhea, nausea, flatulence, back pain, headache, loss of strength and energy, joint pain, pharyngitis, mouth irritation, or painful extremities. Have patient report immediately to prescriber tachycardia, passing out, abnormal heartbeat, or dizziness (HCAHPS).

• Educate patient about signs of a significant reaction (eg, wheezing; chest tightness; fever; itching; bad cough; blue skin color; seizures; or swelling of face, lips, tongue, or throat). Note: This is not a comprehensive list of all side effects. Patient should consult prescriber for additional questions.

Intended Use and Disclaimer: Should not be printed and given to patients. This information is intended to serve as a concise initial reference for health care professionals to use when discussing medications with a patient. You must ultimately rely on your own discretion, experience, and judgment in diagnosing, treating, and advising patients.

Mild Renal Dysfunction: No dose adjustment recommended.
Moderate to Severe Renal Dysfunction: Not recommended

Safety and efficacy have not been established in patients younger than 18 years.

Consult WARNINGS section for additional precautions.

Eliglustat is used to treat Gaucher disease type 1 (a condition in which a certain fatty substance is not broken down normally in the body and builds up in some organs and causes liver, spleen, bone, and blood problems) in certain people. Eliglustat is in a class of medications called enzyme inhibitors. It works by preventing the body from producing the fatty substance so that less of it will build up in the body and cause symptoms.

Eliglustat comes as a capsule to take by mouth. It is usually taken once or twice daily with or without food. Take eliglustat at around the same time(s) every day. Follow the directions on your prescription label carefully, and ask your doctor or pharmacist to explain any part you do not understand. Take eliglustat exactly as directed. Do not take more or less of it or take it more often than prescribed by your doctor.

Swallow the capsules whole with water; do not split, open, chew, dissolve, or crush them.

Eliglustat controls Gaucher disease but does not cure it. Continue to take eliglustat even if you feel well. Do not stop taking eliglustat without talking to your doctor.

This medication may be prescribed for other uses; ask your doctor or pharmacist for more information.

In case of overdose, call the poison control helpline at 1-800-222-1222. Information is also available online at https://www.poisonhelp.org/help. If the victim has collapsed, had a seizure, has trouble breathing, or can't be awakened, immediately call emergency services at 911.

Symptoms of overdose may include the following:

1. dizziness
2. unsteadiness
3. nausea
4. vomiting