Why is this medication prescribed?

Cerdelga (generic name: eliglustat) is prescribed primarily for the treatment of Gaucher disease type 1 in adults. Gaucher disease is a genetic disorder where a specific enzyme deficiency leads to the accumulation of certain substances (glucosylceramide) in various organs and tissues, causing symptoms such as enlarged liver and spleen, bone pain, and fatigue.

Cerdelga works by inhibiting the enzyme glucosylceramide synthase, which helps reduce the accumulation of glucosylceramide in cells affected by Gaucher disease. This treatment helps manage the symptoms and improve quality of life for individuals with Gaucher disease type 1.

How should this medicine be used?

Here’s how Cerdelga should typically be used:

• Dosage: The dosage of Cerdelga is determined individually based on factors such as the patient’s weight, specific enzyme activity levels, and other medical conditions. It’s important to follow the dosage instructions provided by your healthcare provider.
• Administration: Cerdelga is usually taken orally, with or without food. It’s typically taken twice daily.
• Monitoring: During treatment with Cerdelga, regular monitoring of enzyme levels and clinical response is necessary to ensure proper management of Gaucher disease.
• Adherence: It’s important to take Cerdelga exactly as prescribed by your doctor. Missing doses or stopping treatment abruptly can affect its effectiveness.
• Storage: Store Cerdelga at room temperature, away from moisture and heat.
• Special Considerations: Your doctor may adjust the dosage of Cerdelga based on your response to treatment and any side effects you may experience. It’s essential to communicate any concerns or changes in your health to your healthcare provider.

As with any prescription medication, it’s crucial to follow your healthcare provider’s instructions carefully and to ask questions if you have any doubts about how to use Cerdelga.

Other uses for this medicine

Cerdelga (eliglustat) is primarily indicated for the treatment of Gaucher disease type 1 in adults, where it helps manage symptoms by reducing the accumulation of glucosylceramide in cells. This is its approved and primary use. However, there are no other established medical uses for Cerdelga beyond Gaucher disease type 1.

What special precautions should I follow?

When using Cerdelga, there are several special precautions and considerations to keep in mind:

• Hepatic Impairment: Cerdelga may affect liver function, so it is essential to monitor liver enzymes regularly during treatment. Your doctor may adjust the dosage if you have pre-existing liver conditions.
• Drug Interactions: Cerdelga may interact with other medications, potentially affecting their effectiveness or increasing the risk of side effects. Inform your doctor about all medications, supplements, and herbal products you are taking before starting Cerdelga.
• Pregnancy and Breastfeeding: The safety of Cerdelga during pregnancy has not been established. It is recommended to avoid using Cerdelga during pregnancy unless the potential benefit justifies the potential risk to the fetus. It is also not known whether Cerdelga passes into breast milk, so breastfeeding is not recommended during treatment with Cerdelga.
• Genetic Testing: Prior to starting treatment with Cerdelga, genetic testing is recommended to determine if you have a specific genetic mutation (CYP2D6 poor metabolizer status). This can affect how your body metabolizes the medication and help guide dosing adjustments.
• Adverse Reactions: Like any medication, Cerdelga can cause side effects. Common side effects include headache, fatigue, nausea, diarrhea, and upper respiratory tract infections. Serious side effects such as severe allergic reactions or abnormal heart rhythms are rare but possible. Report any unusual symptoms or reactions to your doctor promptly.
• Dietary Considerations: Cerdelga may interact with certain foods or dietary supplements. It’s important to follow any dietary recommendations provided by your doctor or healthcare provider.
• Regular Monitoring: Regular monitoring of your condition, including enzyme levels and symptoms of Gaucher disease, is necessary to assess the effectiveness of Cerdelga and adjust treatment as needed.

Always follow your doctor’s advice and instructions regarding the use of Cerdelga, and do not hesitate to discuss any concerns or questions you may have about its use or potential side effects.

What special dietary instructions should I follow?

• Food Interactions: Cerdelga can be taken with or without food. There are no specific dietary restrictions associated with its use. However, it’s essential to maintain a balanced diet as recommended by your healthcare provider for overall health and wellbeing.
• Grapefruit and Grapefruit Juice: Avoid consuming grapefruit or grapefruit juice while taking Cerdelga, as they may interact with the medication and affect its absorption or metabolism.

What should I do if I forget a dose?

If you miss a dose of Cerdelga, take it as soon as you remember, unless it is almost time for your next scheduled dose. In that case, skip the missed dose and continue with your regular dosing schedule. Do not take a double dose to make up for a missed one.

What side effects can this medication cause?

Cerdelga (eliglustat) can cause various side effects, ranging from mild to severe. It’s important to be aware of these potential side effects and to discuss any concerns with your healthcare provider. Here are some common and less common side effects associated with Cerdelga:

Common Side Effects:

• Headache
• Fatigue
• Nausea
• Diarrhea
• Upper respiratory tract infections
• Musculoskeletal pain (including back pain, extremity pain)
• Abdominal pain
• Dizziness
• Flatulence (gas)
• Insomnia (difficulty sleeping)

Less Common or Serious Side Effects:

• Allergic reactions: Signs may include rash, itching/swelling (especially of the face/tongue/throat), severe dizziness, or trouble breathing.
• Liver problems: Symptoms may include yellowing of the eyes or skin (jaundice), dark urine, persistent nausea or vomiting, stomach/abdominal pain, or unusual tiredness.
• Heart rhythm problems: This may manifest as palpitations or feeling like your heart is racing or pounding.
• Neuropathy (nerve problems): Symptoms can include numbness, tingling, or burning pain in the hands or feet.
• Pancreatitis: Symptoms may include severe abdominal pain, nausea, vomiting, or loss of appetite.
• Low platelet count (thrombocytopenia): This can lead to increased bruising or bleeding.
• Skin reactions: Such as rash, itching, or blisters.
• Kidney problems: Symptoms might include changes in the amount of urine, difficulty urinating, or blood in the urine.
• Infections: Increased susceptibility to infections, including serious infections, has been reported.
• Bone marrow problems: This can lead to decreased production of blood cells (anemia, leukopenia).

If you experience any severe or persistent side effects while taking Cerdelga, it’s crucial to seek medical attention immediately. Your healthcare provider may adjust your dosage or provide supportive care to manage these side effects effectively. It’s also important to inform your doctor of any new symptoms that develop during your treatment with Cerdelga.

What should I know about storage and disposal of this medication?

Storage and Disposal of Cerdelga:

• Storage: Keep Cerdelga tablets in their original container at room temperature, away from moisture and heat. Do not store Cerdelga in the bathroom. Keep it out of reach of children and pets.
• Disposal: Dispose of Cerdelga tablets according to local regulations or guidelines for medication disposal. Do not flush them down the toilet or pour them into a drain unless instructed to do so. Ask your pharmacist or healthcare provider for information on how to properly dispose of unused medication.

In case of emergency/overdose

In case of an overdose or emergency situation related to Cerdelga, it is important to seek immediate medical assistance. Contact your local poison control center (in the United States, call 1-800-222-1222) or go to the nearest emergency room.

What other information should I know?

• Genetic Testing: Before starting Cerdelga treatment, genetic testing is recommended to determine your CYP2D6 metabolizer status. This can influence how your body processes the medication and help guide dosing adjustments.
• Regular Monitoring: Your doctor will monitor your response to Cerdelga through clinical assessments and possibly through laboratory tests to ensure it is effectively managing Gaucher disease type 1.
• Follow-up Visits: Attend all scheduled appointments with your healthcare provider. These visits are important for monitoring your condition and adjusting your treatment plan as needed.
• Medication Compliance: Take Cerdelga exactly as prescribed by your doctor. Do not adjust your dosage or stop taking the medication without consulting your healthcare provider first.
• Travel: If you plan to travel, ensure you have an adequate supply of Cerdelga and any necessary documentation related to your medication. Consider carrying it in your carry-on luggage with the original prescription label.
• Patient Assistance Programs: Depending on your location and circumstances, there may be patient assistance programs available to help with the cost of Cerdelga. Your healthcare provider or pharmacist can provide information on these programs.
• Dietary Considerations: While there are no specific dietary restrictions associated with Cerdelga, avoid consuming grapefruit or grapefruit juice, as they may interact with the medication.
• Side Effects: Be aware of potential side effects of Cerdelga and report any unusual symptoms or reactions to your doctor promptly.

By staying informed about these aspects of Cerdelga treatment, you can effectively manage your condition and optimize the benefits of this medication under the guidance of your healthcare provider.