Eliglustat

Eliglustat is a prescription medication used for long-term treatment of adults with Gaucher disease. 

• Eliglustat is available under the following different brand names: Cerdelga.

Common side effects of Eliglustat include:

• Nausea, 
• Upset stomach, 
• Abdominal pain, 
• Gas, 
• Bloating, 
• Diarrhea, 
• Headache, 
• Back pain, 
• Arm or leg pain, 
• Dizziness, and 
• Weakness

Serious side effects of Eliglustat include:

• Hives, 
• Difficulty breathing, 
• Swelling of the face, lips, tongue, or throat, 
• Severe dizziness, 
• Fainting, 
• Fast or irregular heartbeat, 
• Rash, and
• Itching

Rare side effects of Eliglustat include:

• None 

Seek medical care or call 911 at once if you have the following serious side effects:

• Severe headache, confusion, slurred speech, arm or leg weakness, trouble walking, loss of coordination, feeling unsteady, very stiff muscles, high fever, profuse sweating, or tremors;
• Serious eye symptoms such as sudden vision loss, blurred vision, tunnel vision, eye pain or swelling, or seeing halos around lights;
• Serious heart symptoms include fast, irregular, or pounding heartbeats; fluttering in the chest; shortness of breath; sudden dizziness, lightheadedness, or passing out.

This is not a complete list of side effects and other serious side effects or health problems that may occur because of the use of this drug. Call your doctor for medical advice about serious side effects or adverse reactions. You may report side effects or health problems to FDA at 1-800-FDA-1088.

Adult dosage

Capsule

• 84 mg

Gaucher Disease

Adult dosage

• Dose based on the patient's CYP2D6 metabolizer status.
• CYP2D6 EM or IM: 84 mg orally two times a day
• CYP2D6 PM: 84 mg orally once a day

Dosage Considerations – Should be Given as Follows: 

• See “Dosages”

If your medical doctor is using this medicine to treat your pain, your doctor or pharmacist may already be aware of any possible drug interactions and may be monitoring you for them. Do not start, stop, or change the dosage of any medicine before checking with your doctor, health care provider, or pharmacist first.

• Eliglustat has severe interactions with at least 63 other drugs.
• Eliglustat has serious interactions with at least 159 other drugs.
• Eliglustat has moderate interactions with at least 198 other drugs.
• Eliglustat has minor interactions with the following drugs.
  • acetazolamide
  • anastrozole
  • cyclophosphamide
  • larotrectinib

This information does not contain all possible interactions or adverse effects. Visit the RxList Drug Interaction Checker for any drug interactions. Therefore, before using this product, tell your doctor or pharmacist about all your products. Keep a list of all your medications with you and share this information with your doctor and pharmacist. Check with your healthcare professional or doctor for additional medical advice, health questions, or concerns.

Contraindications

• Extensive CYP2D6 metabolizers
  • Coadministration with strong or moderate CYP2D6 inhibitor AND strong or moderate CYP3A inhibitor
  • Moderate or severe hepatic impairment
  • Mild hepatic impairment and taking strong or moderate CYP2D6 inhibitors.
• Intermediate CYP2D6 metabolizers
  • Coadministration with strong or moderate CYP2D6 inhibitor AND strong or moderate CYP3A inhibitor
  • Coadministration with strong CYP3A inhibitors
  • Any degree of hepatic impairment
• Poor CYP2D6 metabolizers
  • Coadministration with strong CYP3A inhibitors
  • Any degree of hepatic impairment

Effects of drug abuse

• None

Short-Term Effects

• See “What Are Side Effects Associated with Using Eliglustat?”

Long-Term Effects

• See “What Are Side Effects Associated with Using Eliglustat?”

Cautions

• Increases in ECG intervals (PR, QTc, and QRS) occur at substantially elevated eliglustat plasma concentrations; avoid use in patients with pre-existing cardiac conditions (. g, CHF, recent acute MI, bradycardia, heart block, ventricular arrhythmia), long QT syndrome, and in combination with class IA (.g, quinidine, procainamide) and class III (.g, amiodarone, sotalol) antiarrhythmics
• Drug interaction overview
  • Eliglustat inhibits P-gp and CYP2D6; coadministration with P-gp or CYP2D6 substrates may result in increased concentrations of the concomitant drug.
  • Use of eliglustat is contraindicated, to be avoided, or may require dosage adjustment depending on the concomitant drug and CYP2D6 metabolizer status (ie, EM, IM, PM)
  • CYP2D6 and CYP3A substrate; drugs that inhibit CYP2D6 and CYP3A metabolism may significantly increase eliglustat systemic exposure and result in prolongation of the PR, QTc, and/or QRS cardiac intervals
  • See Dosage Modifications

Pregnancy and Lactation

• Available data on use in pregnant women include 20 pregnancies that occurred during the clinical development program and a small number of postmarketing case reports.
• These data are not sufficient to assess drug-associated risks of major birth defects, miscarriage, or adverse maternal or fetal outcomes.
• Disease-associated risk
  • Women with Gaucher disease type 1 have an increased risk of spontaneous abortion, especially if disease symptoms are not treated and controlled preconception and during a pregnancy
  • Pregnancy may exacerbate existing Gaucher disease type 1 symptoms or result in new disease manifestations
  • Gaucher disease type 1 manifestation may lead to adverse pregnancy outcomes including, hepatosplenomegaly which can interfere with the normal growth of pregnancy, and thrombocytopenia which can lead to increased bleeding and possible hemorrhage.
• Lactation
  • There are no human data available on the presence of eliglustat in human milk, the effects on the breastfed infant, or the effects on milk production.
  • Eliglustat and its metabolites were present in the milk of lactating rats.