Cerdelga (eliglustat) (Eliglustat CAPSULE) 84 mg/1

Cerdelga (eliglustat) is an oral glucosylceramide synthase inhibitor approved by the FDA for the treatment of Gaucher disease type 1 in adults. It received orphan drug designation and represents an important therapeutic option in the rare genetic disorder category. This medication falls within the neurology and genetics therapeutic areas, offering substrate reduction therapy for this lysosomal storage disorder.

Strength

84 mg/1

Pack Size

4 BLISTER PACK in 1 CARTON (58468-0220-1) / 14 CAPSULE in 1 BLISTER PACK

Manufacturer / TM Owner

Sanofi Genzyme

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Pack/s

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Indications & Clinical Uses

Cerdelga is indicated for the treatment of Gaucher disease type 1 (GD1), a rare inherited lysosomal storage disorder caused by deficiency of the enzyme glucocerebrosidase. The disease primarily affects adults with non-neuronopathic manifestations, including hepatosplenomegaly, bone disease, anemia, and thrombocytopenia. Cerdelga works through substrate reduction therapy by inhibiting glucosylceramide synthase, thereby decreasing the accumulation of glucosylceramide in macrophages and other tissues. The target patient population includes adults with confirmed Gaucher disease type 1 who require disease-modifying therapy. By reducing substrate accumulation, Cerdelga helps alleviate visceral organomegaly, improves hematologic parameters, and reduces bone complications. The drug is particularly beneficial for patients seeking an oral alternative to intravenous enzyme replacement therapy, offering improved convenience and quality of life while maintaining therapeutic efficacy in managing disease progression.

Side Effects

Common adverse effects of Cerdelga include diarrhea, fatigue, headache, upper respiratory tract infections, and joint pain. Serious adverse events, though less frequent, may include cardiac arrhythmias, particularly in patients with baseline QT prolongation or those taking CYP3A4 inhibitors. Hepatic enzyme elevations and peripheral neuropathy have been reported. Patients require baseline electrocardiogram (ECG) assessment and periodic monitoring, particularly if CYP3A4 inhibitor co-medications are introduced. Regular laboratory monitoring including liver function tests, complete blood counts, and cardiac assessment is recommended. Patients should report symptoms such as palpitations, syncope, or neurological changes immediately to their healthcare provider.

Precautions & Warnings

Cerdelga is contraindicated in patients with significant cardiac conduction abnormalities or those taking strong CYP3A4 inhibitors due to increased QT prolongation risk. Baseline ECG is mandatory before initiating therapy. Use with caution in patients with hepatic or renal impairment; dose adjustment may be necessary. Pregnancy category C—use only if benefits outweigh risks; breastfeeding is not recommended. Pediatric use has not been established; safety and efficacy in children remain unknown. Patients with cardiac risk factors, electrolyte abnormalities, or family history of sudden cardiac death require careful evaluation. Regular cardiac monitoring is essential, particularly when initiating therapy or adjusting concomitant medications.

Avoid Interactions

Cerdelga is metabolized by CYP3A4 and CYP2D6, making it susceptible to significant drug interactions. Strong CYP3A4 inhibitors (ketoconazole, ritonavir, clarithromycin) are contraindicated due to increased eliglustat exposure and QT prolongation risk. Moderate CYP3A4 inhibitors require dose reduction. CYP2D6 inhibitors may increase eliglustat levels. Grapefruit juice should be avoided as it inhibits CYP3A4. Patients taking antiarrhythmic agents or other QT-prolonging medications require enhanced cardiac monitoring. Concurrent use with certain antiretrovirals or antifungals necessitates careful clinical assessment and potential dose adjustments.

Frequently Asked Questions

Cerdelga is used to treat Gaucher disease type 1, a rare inherited lysosomal storage disorder. It works by reducing the accumulation of glucosylceramide in the body, thereby alleviating symptoms such as enlarged liver and spleen, bone disease, anemia, and low platelet counts. Cerdelga is specifically indicated for adults with confirmed Gaucher disease type 1.

Cerdelga is administered orally as a capsule, typically at a dose of 84 mg twice daily for most patients. The capsules should be swallowed whole with water and can be taken with or without food. Dose adjustments may be necessary based on CYP2D6 metabolizer status and concomitant medications.

Common side effects include diarrhea, fatigue, headache, upper respiratory tract infections, and joint pain. Most side effects are mild to moderate in severity. Serious but less common adverse effects may include cardiac arrhythmias and liver enzyme elevations, requiring medical monitoring.

Cerdelga is manufactured by Sanofi Genzyme, a subsidiary of Sanofi specializing in rare disease treatments and genetic disorders.

Yes, Cerdelga received orphan drug designation from the FDA due to its indication for Gaucher disease type 1, a rare genetic disorder affecting fewer than 200,000 individuals in the United States. This designation provides regulatory incentives and market exclusivity to support development of treatments for rare diseases.

Published by

GNH India Pharmaceuticals Limited

Licensed pharmaceutical supplier

Last updated: 17 Jun 2026

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