What is Larglustat?

People with Gaucher disease lack an enzyme called glucocerebrosidase, causing a fatty substance called glucosylceramide to accumulate in the body. This build-up leads to various symptoms and complications, including: Enlarged liver and spleen Low blood cell counts (anemia and thrombocytopenia) bone pain and other skeletal problems. Larglustat (Eliglustat) is an oral medication used for the long-term treatment of Type 1 Gaucher disease in adults. It is a type of therapy called a substrate reduction therapy (SRT).

How Larglustat Works?

Eliglustat works by acting as a substrate reduction therapy (SRT). Instead of replacing the deficient enzyme, it inhibits glucosylceramide synthase, the enzyme responsible for producing glucosylceramide. By blocking the production of this fatty substance, eliglustat prevents its harmful build-up in organs like the liver and spleen, and in the bone marrow.

Who Should Take Larglustat?

Larglustat is indicated for the long-term treatment of adult patients with Gaucher disease type 1 who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs) as detected by an FDA-cleared test.

Product Specifications

Dosage and Administration

Patient Selection:

Select patients using an FDA-cleared test for determining CYP2D6 genotype.

Recommended Dosage Based on CYP2D6 Metabolizer Status:

• EMs and IMs: 84 mg orally twice daily.
• PMs: 84 mg orally once daily.

Administration:

• Swallow capsules whole; do not crush, dissolve, or open capsules.
• Avoid eating grapefruit or drinking grapefruit juice.

Administration Guidelines:

• Swallow capsules whole, preferably with water; do not crush, dissolve, or open the capsules.
• Larglustat can be taken with or without food.
• Avoid the consumption of grapefruit or grapefruit juice (strong CYP3A inhibitors) with Larglustat.
• If a dose of Larglustat is missed, take the prescribed dose at the next scheduled time; do not double the next dose.
• For patients currently treated with imiglucerase, velaglucerase alfa, or taliglucerase alfa, Larglustat may be administered 24 hours after the last dose of the previous enzyme replacement therapy (ERT).

Monitoring Requirements

Gaucher disease stability: Regular checks of hemoglobin, platelet counts, and organ volumes to confirm the drug's ongoing effectiveness.

Cardiac safety: Periodic ECGs are needed to monitor for any cardiac interval changes, particularly in patients with pre-existing heart conditions or those taking interacting medications.

Adverse events: Monitoring for side effects like headache, fatigue, or gastrointestinal issues, with particular attention to potential cardiac symptoms.

Drug interactions: Continued screening for new medications that could interfere with eliglustat's metabolism through the CYP2D6 and CYP3A pathways.

Biomarker analysis: Tracking biomarkers such as glucosylsphingosine and chitotriosidase to confirm therapeutic response.

Safety Information

Common Side Effects

Most common adverse reactions (≥10%) are: fatigue, headache, nausea, diarrhoea, back pain, pain in extremities, and upper abdominal pain.

Serious Warnings

ECG Changes and Potential for Cardiac Arrhythmias: Not recommended in patients with pre-existing cardiac disease, long QT syndrome, and concomitant use of Class IA and Class III antiarrhythmic.

Drug Interactions

Eliglustat has significant drug interactions, particularly with CYP2D6 and CYP3A4 inhibitors (like fluoxetine, paroxetine, and ketoconazole), which can increase eliglustat levels and risk cardiac events, making co-administration contraindicated in some cases. Strong CYP3A4 inducers (like rifampin and St. John's Wort) can decrease eliglustat levels and reduce its effectiveness.

Storage Requirements

• Store at room temperature between 15-30°C (59-86°F)

Product Pricing and Availability

• Available Pack Sizes: 56 capsule per boxs
• Pricing Information:1,69,500 per box
• Where to Purchase: Call customer care number: 7337585050
• Last Updated: November 27, 2025

Frequently Asked Questions (FAQ)

FDA Approval and Clinical Evidence

Eliglustat was first approved by the U.S. Food and Drug Administration (FDA) on August 19, 2014, for the long-term treatment of adults with Gaucher disease type 1 (GD1). The approval was based on clinical evidence from multiple trials demonstrating its effectiveness and safety

Clinical evidence

For treatment-naïve patients (ENGAGE trial)

• Study design: A randomized, double-blind, placebo-controlled Phase 3 trial, known as ENGAGE, was conducted with 40 treatment-naïve adult GD1 patients.

Key finding:

After 9 months, patients on eliglustat showed significant improvements compared to the placebo group, including:

• Greater reduction in spleen volume.
• Improved liver volume
• Increased blood platelet count.
• Higher hemoglobin levels

Long-term Data

The open-label extension phase of the ENGAGE trial demonstrated that these improvements continued for up to 4.5 years.

For Previously Treated Patients (ENCORE Trial)

Study design: The ENCORE trial was a randomized, open-label, non-inferiority Phase 3 study comparing eliglustat to the enzyme replacement therapy (ERT) imiglucerase in 160 GD1 patients whose disease was stable on ERT.

Key finding: Eliglustat was non-inferior to imiglucerase in maintaining disease stability after one year. The drug was successful in keeping platelet counts, hemoglobin levels, and organ volumes stable.

Long-term data: Continued treatment with eliglustat for up to four years in the ENCORE extension phase maintained the clinical stability of patients.

Important Reminders

• Take Larglustat exactly as prescribed by your healthcare provider
• Do not adjust your dose without medical supervision
• Keep all scheduled appointments for monitoring
• Report any new symptoms or concerns promptly
• Store medication properly and check expiration dates
• Ensure adequate supply and refill prescriptions in advance
• Educate family members and caregivers about your condition and emergency signs