| Common Brands | Cerdelga |
|---|---|
|
Drug Class |
Glucosylceramide synthase inhibitor |
|
Controlled Substance Classification |
Not a controlled medication |
|
Generic Status |
No Lower-cost generic available |
|
Availability |
Prescription only |
Cerdelga is the brand name for the generic drug eliglustat. It is an oral drug that treats a rare genetic disorder called Gaucher disease. This disease results from a deficiency of the enzyme glucocerebrosidase, causing the accumulation of a substance known as glucocerebroside in certain organs, such as the spleen, liver, and bone marrow. Gradually, this causes serious damage to the mentioned organs and subsequently results in some symptoms, including splenomegaly and hepatomegaly, bone pain, and anemia. Cerdelga inhibits the synthesis of glucocerebroside, therefore preventing its accumulation.
Since the FDA approval in 2014, Cerdelga provided patients with Gaucher disease with an alternative treatment modality, which is especially crucial for those candidates who are not suitable for replacement enzyme therapies or prefer an oral treatment. In this regard, Cerdelga offers an alternative that can effectively manage the symptoms of Gaucher disease and further improve quality of life.
The capsule of Cerdelga should be orally ingested, taken with or without food. It is important for the patient to follow his physician’s instruction in terms of dosing and administration. Swallow the capsule with a glass of water, do not crush, chew, or break it.
It is crucial for patients to strictly adhere to the dosage regimen as prescribed and consult their healthcare provider if they notice any side effects or issues with the medication. Doses should not be missed; however, if a dose is missed, patients should take it as soon as they remember unless it is close to the time for their next dose.
It works by interfering with a protein called glucosylceramide synthase or UGCG. This product catalyzes the formation of glucocerebroside, a product that is accumulated within cells of the affected patients of Gaucher disease due to the deficiency in glucocerebrosidase.
Through the inhibition of glucocerebroside synthesis, Cerdelga reduces the accumulation of this material in numerous organs such as the liver, spleen, and bone marrow. This helps prevent the harmful effects of Gaucher disease, such as organ enlargement, pain, and other related complications.
The drug works at a molecular level by targeting the pathway that produces glucocerebroside, hence why it is considered an effective treatment for managing Gaucher disease. Cerdelga does not replace the missing enzyme (glucocerebrosidase) but addresses the buildup of its substrate, glucocerebroside.
Cerdelga, like any medication, can cause side effects. Some of the most common side effects associated with Cerdelga include:
Less common, but more severe side effects may include:
Hepatic impairment: Liver enzyme levels must be monitored to avoid complications during treatment. White blood cells and platelet count are usually affected.
Cardiovascular disorder: Some cardiac disorders may also occur particularly in patients already with cardiac disorders. The patient should seek the opinion of their doctor in case of any severe side effects or symptoms of concern.
Before starting treatment with Cerdelga, patients should inform their healthcare providers about any existing health conditions, especially:
Liver disease: Cerdelga may cause liver problems, and liver function should be monitored regularly.
Heart issues: Cerdelga can affect the heart, especially in patients with heart conditions or those on certain medications.
Pregnancy and breastfeeding: The safety of Cerdelga in pregnancy or breastfeeding is not determined, and it should be started only when the potential benefits exceed the risks.
Genetic testing: Due to the fact that Cerdelga undergoes extensive metabolism by particular liver enzymes, patients must undergo genetic testing so that they can metabolize the drug appropriately.
Cerdelga is administered according to the patient’s health status, genetic predispositions, as well as the severity of their Gaucher disease. The medication will most likely be in the form of an oral capsule, and the starting dose is usually 84 mg taken twice daily, though sometimes, it can be lower if the patient is a poor metabolizer of the drug.
Cerdelga may interact with other drugs that may either decrease its effectiveness or increase the risk of side effects. For instance, drugs that affect the liver enzymes involved in metabolizing Cerdelga, such as cytochrome P450 enzymes, can cause a change in the drug’s blood levels. Some of the drugs that may interact with Cerdelga include:
Other drugs that affect the activity of liver enzymes
Patients should be advised to inform their healthcare providers about all medications, including over-the-counter drugs and supplements, in order to avoid harmful interactions.
The dosage to be administered as recommended for Cerdelga would depend on certain factors, particularly the patient’s CYP2D6 genetic profile. Common starting doses among adults are the following:
The dose might be adjusted to the patient’s response and tolerance to the medication, and it is closely followed by the practitioner with respect to liver function and blood cell count, among others.
The drug Cerdelga is strictly a prescription one. Only when a patient sees a healthcare provider and goes through the condition would the health professional run appropriate tests and see whether Cerdelga is suitable. Follow-up check-ups will ensure that no unwanted side effects result from this prescription and if indeed it’s effectively working for them.
Answer: Cerdelga (eliglustat) is an oral medication used to treat certain adults with type 1 Gaucher disease, a rare genetic disorder that affects fat metabolism.
Answer: Cerdelga is a glucosylceramide synthase inhibitor that reduces the production of fatty substances that accumulate in cells due to Gaucher disease.
Answer: Cerdelga is approved for adults with type 1 Gaucher disease who are extensive or intermediate CYP2D6 metabolizers, based on genetic testing.
Answer: Gaucher disease type 1 is a genetic disorder caused by the deficiency of the enzyme glucocerebrosidase, leading to fat buildup in organs and bones.
Answer: Yes, patients must undergo CYP2D6 genotyping to determine if they are suitable for treatment with Cerdelga.
Answer: Cerdelga is taken orally, usually twice daily, with or without food, as prescribed by a healthcare provider.
Answer: Common side effects include fatigue, nausea, diarrhea, headache, and back pain.
Answer: The safety of Cerdelga in pregnancy has not been well established, so it should only be used if clearly needed and under medical supervision.
Answer: No, Cerdelga is not a cure, but it helps manage and control symptoms of type 1 Gaucher disease.
Answer: No, Cerdelga is only available as a branded medication by Sanofi Genzyme.
Answer: If you miss a dose, take it as soon as you remember. If it’s close to your next dose, skip the missed one—do not double up.
Answer: Cerdelga may interact with other drugs, especially those affecting CYP2D6 and CYP3A enzymes. Always inform your doctor of all medications you’re taking.
Answer: Cerdelga is an oral substrate reduction therapy (SRT), whereas ERT is given by intravenous infusion to replace the missing enzyme.
Answer: It may take several months to observe improvements in symptoms, depending on individual response and disease severity.
Answer: No, Cerdelga is not classified as a controlled substance.
Answer: Cerdelga is currently approved only for adults and is not recommended for pediatric patients.
Answer: Avoid grapefruit and grapefruit juice, as they can increase Cerdelga levels and the risk of side effects.
Answer: Store Cerdelga at room temperature, away from moisture and heat.
Answer: Yes, the manufacturer may offer patient support programs or copay assistance for eligible individuals.
Answer: No, do not stop Cerdelga without consulting your doctor. Continuous treatment is necessary to control disease progression.
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