Generic Name: eliglustat
Date of Approval: August 19, 2014
Company: Genzyme Corporation

Treatment for: Gaucher Disease Type 1

FDA Approves Cerdelga

The U.S. Food and Drug Administration (FDA) has approved Cerdelga (eliglustat), a glucosylceramide synthase inhibitor administered orally for the long-term treatment of adults with Gaucher disease type 1. It is indicated for use in patients who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs) as detected by an FDA-cleared test.

Gaucher disease is an inherited condition caused by deficiency of the enzyme glucocerebrosidase, which causes an accumulation of glucosylceramide in cells and tissues of the spleen, liver, kidneys, lungs, brain, and bone marrow. Symptoms include liver and spleen enlargement, anemia, low blood platelets and bone problems. Cerdelga works by reducing the production of glucosylceramide.

The most common side effects are fatigue, headache, nausea, diarrhea, back pain, pain in extremities, and upper abdominal pain.

Cerdelga Medication Guide

Read the Medication Guide that comes with your prescription before you start using it and each time you get a refill. There may be new information. This Medication Guide does not take the place of talking to your healthcare provider about your medical condition or treatment.

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Important information about Cerdelga

Cerdelga can affect the way other medicines work and vice versa, so taking it with other medicines or herbal supplements may cause an increased risk of side effects.

Especially tell your doctor if you take:

  • St. John’s Wort (Hypericum perforatum)
  • Medicine for:
    • Fungal infections
    • Tuberculosis
    • Seizures
    • Heart conditions or high blood pressure
    • Depression or other mental health problems

If you take any medicines for the conditions listed above, your doctor may need to prescribe a different medicine, or change your doses. Tell your doctor about any new medicines before you start taking them.

What is Cerdelga?

Cerdelga is a prescription medicine used for the long-term treatment of Gaucher disease type 1 (GD1) in adults. It is not used in certain people with Gaucher disease type 1. Your doctor will perform a test to make sure that it is right for you. It is not known if it is safe and effective in children.

Before taking Cerdelga

Tell your doctor about all of your medical conditions, including if you:

  • have heart problems, including a condition called long QT syndrome
  • have a history of a heart attack
  • have kidney or liver problems
  • are pregnant or planning to become pregnant. It is not known if this medicine will harm your unborn baby.
  • are breastfeeding or planning to breastfeed. It is not known if this medicine passes into your breast milk. You and your doctor will decide if you should take Cerdelga or breastfeed. You should not do both.

Tell your doctor about all of the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. See Important information about Cerdelga

How should I take Cerdelga?

  • Take exactly as your doctor tells you to take it.
  • Your doctor may change your dose if needed.
  • Take the capsules whole, preferably with water. Do not open, crush, or dissolve capsules before swallowing.
  • Capsules can be taken with or without food.
  • If you miss a dose, take the next dose at the usual time. Do not take two doses at the same time.
  • If you take too much (overdose), call your doctor or go to the nearest hospital emergency room right away.

What should I avoid while taking Cerdelga?

Avoid eating or drinking grapefruit products. Grapefruit products can increase the amount of the drug in your body.

Cerdelga side effects

See Important information about Cerdelga

  • Cerdelga, used with certain other medicines, may cause changes in the electrical activity of your heart (ECG changes) and irregular heart beat (arrhythmias). Tell your doctor if you have new symptoms such as palpitations, fainting, or dizziness.

The most common side effects include: tiredness, headache, nausea, diarrhea, and pain in the arms, legs, back, or stomach (abdomen).

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

These are not all the possible side effects. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

See also: Side effects (in more detail)

How should I store Cerdelga?

  • Store at room temperature between 68°F to 77 °F (20°C to 25 °C).
  • Keep all medicines out of reach of children.

General information

Medicines are sometimes prescribed for purposes other than those listed in a Medication Guide. Do not use this medicine for a condition for which it was not prescribed. Do not give it to other people, even if they have the same symptoms you have. It may harm them. If you would like more information, talk with your doctor. You can ask your doctor or pharmacist for information that is written for health professionals.

What are the ingredients?

Active ingredient: eliglustat

Inactive ingredients: microcrystalline cellulose, lactose monohydrate, hypromellose, glyceryl behenate, gelatin, candurin silver fine, yellow iron oxide, and FD&C blue 2