Eliglustat — Membership Pricing

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About Eliglustat

Eliglustat is a specialized medication used to treat Gaucher disease type 1, a rare genetic disorder. As a critical treatment, understanding its role and managing its cost is essential. TheDrugClub is dedicated to helping members access vital prescriptions like eliglustat, offering potential savings and support to make your healthcare more affordable.

Eliglustat is specifically approved for the long-term treatment of adults with Gaucher disease type 1 (GD1). GD1 is a rare, inherited metabolic disorder where the body lacks an enzyme called glucocerebrosidase. This enzyme normally breaks down a fatty substance called glucocerebroside. Without enough of it, glucocerebroside builds up in various organs, including the spleen, liver, and bone marrow, leading to symptoms like an enlarged spleen and liver, anemia, bone pain, and fatigue. Eliglustat helps manage these severe symptoms and prevent further progression of the disease.

Eliglustat works as a substrate reduction therapy (SRT). Instead of replacing the missing enzyme, it reduces the amount of glucocerebroside the body produces in the first place. By inhibiting the enzyme glucosylceramide synthase, eliglustat slows down the synthesis of glucocerebroside. This reduction in the production of the fatty substance helps prevent its accumulation in cells, thereby alleviating the symptoms and progression of Gaucher disease type 1. It's an oral medication, offering a convenient alternative to enzyme replacement therapy (ERT) which typically requires intravenous infusions.

Due to its specialized nature and the rarity of the condition it treats, eliglustat is a high-cost medication. Without insurance or significant discounts, a monthly supply can range from several thousand to tens of thousands of dollars, placing a considerable financial burden on patients. These costs can fluctuate based on the pharmacy, dosage, and location; for example, a pharmacy in

The information above is for educational purposes only and does not constitute medical advice. Consult your healthcare provider before making medication decisions.

What Eliglustat is approved for (FDA label)

CERDELGA is indicated for the long-term treatment of adult patients with Gaucher disease type 1 (GD1) who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs) as detected by an FDA-cleared test [see Dosage and Administration (2.1) ] . CERDELGA is a glucosylceramide synthase inhibitor 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. ( 1 ) Limitations of Use : CYP2D6 ultra-rapid metabolizers may not achieve adequate concentrations of CERDELGA to achieve a therapeutic effect. ( 1 ) A specific dosage cannot be recommended for CYP2D6 indeterminate metabolizers. ( 1 ) Limitations of Use : Patients who are CYP2D6 ultra-rapid metabolizers (URMs) may not achieve adequate concentrations of CERDELGA to achieve a therapeutic effect [see Clinical Studies (14) ] . A specific dosage cannot be recommended for those patients whose CYP2D6 genotype cannot be determined (indeterminate metabolizers) [see Clinical Studies (14) ] .

Source: Read the full FDA label on DailyMed. Label effective date: March 3, 2024.

Excerpt from the FDA-approved prescribing information. This is not medical advice; talk to your doctor or pharmacist.

Dosage forms and strengths for Eliglustat (FDA label)

Capsules: 84 mg of eliglustat is in a capsule with a pearl blue-green opaque cap and pearl white opaque body imprinted with "GZ02" in black. Capsules: 84 mg eliglustat

Source: FDA prescribing information (DailyMed). Set ID: 819f828a-b888-4e46-83fc-94d774a28a83. SPL document ID: e71b0b39-5a29-4e38-8500-55b6d1f48543. Label effective date: March 3, 2024. Read the full label.

Excerpt from the FDA-approved prescribing information. This is not medical advice; talk to your doctor or pharmacist.

Adverse reactions for Eliglustat (FDA label)

Most common adverse reactions (≥10%) are: fatigue, headache, nausea, diarrhea, back pain, pain in extremities, and upper abdominal pain. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Genzyme Corporation at 1-800-745-4447 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.

Source: FDA prescribing information (DailyMed). Set ID: 819f828a-b888-4e46-83fc-94d774a28a83. SPL document ID: e71b0b39-5a29-4e38-8500-55b6d1f48543. Label effective date: March 3, 2024. Read the full label.

Excerpt from the FDA-approved prescribing information. This is not medical advice; talk to your doctor or pharmacist.

Contraindications for Eliglustat (FDA label)

CERDELGA is contraindicated in the following patients based on CYP2D6 metabolizer status due to the risk of cardiac arrhythmias from prolongation of the PR, QTc, and/or QRS cardiac intervals.

Source: FDA prescribing information (DailyMed). Set ID: 819f828a-b888-4e46-83fc-94d774a28a83. SPL document ID: e71b0b39-5a29-4e38-8500-55b6d1f48543. Label effective date: March 3, 2024. Read the full label.

Excerpt from the FDA-approved prescribing information. This is not medical advice; talk to your doctor or pharmacist.

Warnings and precautions for Eliglustat (FDA label)

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 antiarrhythmics. ( 5.1 ) 5.1 ECG Changes and Potential for Cardiac Arrhythmias CERDELGA is predicted to cause increases in ECG intervals (PR, QTc, and QRS) at substantially elevated eliglustat plasma concentrations and may increase the risk of cardiac arrhythmias. Use of CERDELGA is contraindicated, to be avoided, or requires dosage adjustment in patients taking CYP2D6 or CYP3A inhibitors, depending on CYP2D6 metabolizer status, type of inhibitor, or degree of hepatic impairment [see Dosage and Administration (2.3) , Contraindications (4) , Drug Interactions (7.1) ] . Use of CERDELGA in patients with pre-existing cardiac conditions has not been studied during clinical trials.

Source: FDA prescribing information (DailyMed). Set ID: 819f828a-b888-4e46-83fc-94d774a28a83. SPL document ID: e71b0b39-5a29-4e38-8500-55b6d1f48543. Label effective date: March 3, 2024. Read the full label.

Excerpt from the FDA-approved prescribing information. This is not medical advice; talk to your doctor or pharmacist.

Drug interactions for Eliglustat (FDA label)

See Full Prescribing Information for a list of clinically significant drug interactions. ( 7.1 , 7.2 ) 7.1 Effect of Other Drugs on CERDELGA Coadministration of CERDELGA with: CYP2D6 or CYP3A inhibitors may increase eliglustat concentrations which may increase the risk of cardiac arrhythmias from prolongation of the PR, QTc, and/or QRS cardiac interval [see Warnings and Precautions (5.1) , Clinical Pharmacology (12.3) ] . strong CYP3A inducers decrease eliglustat concentrations which may reduce CERDELGA efficacy [see Clinical Pharmacology (12.3) ] . See Table 5 for prevention and management of interactions with drugs affecting CERDELGA. Use of CERDELGA is contraindicated, to be avoided, or may require dosage adjustment depending on the concomitant drug and CYP2D6 metabolizer status [see Dosage and Administration (2.2 , 2.3) , Contraindications (4) , Drug Interactions (7.1) ] . Table 5: Prevention and Management Strategies of Drug Interactions Affecting CERDELGA Based on CYP2D6 Metabolizer Status and

Source: FDA prescribing information (DailyMed). Set ID: 819f828a-b888-4e46-83fc-94d774a28a83. SPL document ID: e71b0b39-5a29-4e38-8500-55b6d1f48543. Label effective date: March 3, 2024. Read the full label.

Excerpt from the FDA-approved prescribing information. This is not medical advice; talk to your doctor or pharmacist.