Cerdelga

Cerdelga is a prescription medication used for long-term treatment of adults with Gaucher disease type 1, which is an inherited enzyme deficiency that can affect most of the body’s organs and tissues.

Cerdelga is a glucosylceramide synthase inhibitor. It works by replacing the enzyme that is not naturally produced by the body in Gaucher disease.

This medication comes in oral capsule form and is take one to two times a day, with or without food.

Do not chew, divide, or break Cerdelga capsules. Swallow the capsules whole.

Common side effects of Cerdelga include fatigue, headache, nausea, diarrhea, back pain, pain in the extremities, and upper abdominal pain. Cerdelga can also cause dizziness. Do not drive or operate heavy machinery until you know how Cerdelga affects you.

Cerdelga is a prescription medication used for long-term treatment of adults with Gaucher disease type 1, depending on their CYP2D6 metabolizer status. A CYP2D6 genotype test is required before therapy is initiated.

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

• Genzyme Corporation

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

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

This is not a complete list of Cerdelga 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.

Tell your doctor about all the medicines you take, including prescription and non-prescription medicines, vitamins, and herbal supplements. Especially tell your doctor if you take:

• CYP2D6 inhibitors such as paroxetine (Paxil), terbinafine (Lamisil), ketoconazole (Nizoral), or fluconazole (Diflucan)
• CYP3A4 inhibitors such as ranitidine (Zantac)
• CYP3A4 inducers such as rifampin (Rifadin), carbamazepine (Tegretol, Carbatrol), phenobarbital, phenytoin (Dilantin, Phenytek), and St. John’s Wort.
• CYP2D6 substrates such as metoprolol (Toprol), tricyclic antidepressants including nortriptyline (Pamelor), amitriptyline, and imipramine (Tofranil), perphenazine, and chlorpromazine

This is not a complete list of Cerdelga drug interactions. Ask your doctor or pharmacist for more information.

Cerdelga has the potential to cause cardiac arrhythmias and increased ECG intervals.

Do not take Cerdelga if you:

• are allergic Cerdelga or to any of its ingredients
• have pre-existing cardiac disease or long QT syndrome
• are taking drugs that are strong inhibitors of CYP3A4

Grapefruit and grapefruit juice may interact with Cerdelga and lead to potentially dangerous effects. Discuss the use of grapefruit products with your doctor.

Before taking Cerdelga, tell your doctor about all of your medical conditions. Especially tell your doctor if you:

• are allergic to Cerdelga or to any of its ingredients
• have or have had cardiac disease
• have liver problems
• have kidney problems
• are pregnant or breastfeeding

Tell your doctor about all the medicines you take including prescription and non-prescription medicines, vitamins, and herbal supplements.

Tell your doctor if you are pregnant or plan to become pregnant.

The FDA categorizes medications based on safety for use during pregnancy. Five categories - A, B, C, D, and X, are used to classify the possible risks to an unborn baby when a medication is taken during pregnancy.

Cerdelga falls into category C. In animal studies, pregnant animals were given this medication and had some babies born with problems. No well-controlled studies have been done in humans. Therefore, this medication may be used if the potential benefits to the mother outweigh the potential risks to the unborn child.

Tell your doctor if you are breastfeeding or plan to breastfeed.

It is not known if Cerdelga crosses into human milk. Because many medications can cross into human milk and because of the possibility for serious adverse reactions in nursing infants with use of this medication, a choice should be made whether to stop nursing or stop the use of this medication. Your doctor and you will decide if the benefits outweigh the risk of using Cerdelga.

Take Cerdelga exactly as prescribed.

Cerdelga comes in capsule form and is taken once or twice daily.  Do not chew, divide, or break Cerdelga capsules. Swallow capsules whole.

If you miss a dose, take the missed dose as soon as you remember. If it is almost time for the next dose, skip the missed dose and take your next dose at the regular time. Do not take two doses of Cerdelga at the same time.

Take this medication exactly as prescribed by your doctor. Follow the directions on your prescription label carefully.

The dose your doctor recommends may be based on the following:

• the condition being treated
• your CYP 2D6 metabolizer status
• other medications you are taking

The recommended dose of Cerdelga for the treatment of Gaucher disease type 1 is 84 mg twice daily in extensive metabolizers and intermediate metabolizers and 84 mg once daily in poor metabolizers.

If you take too much Cerdelga, call your healthcare provider or local Poison Control Center, or seek emergency medical attention right away.

Store Cerdelga at room temperature.

Keep this and all medicines out of the reach of children.