Eliglustat (Cerdelga) is a glucosylceramide synthase inhibitor that received its first approval in the United States in 2014. As a targeted therapy for Type 1 Gaucher disease, it acts as a substrate reduction therapy by decreasing the production of glucosylceramide (GL-1).

What Kind of Drug Is Eliglustat (Cerdelga)?

Indication Scope

Eliglustat is indicated for the long-term treatment of adult patients with Type 1 Gaucher disease (GD1).

However, these patients must be confirmed as CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs) through FDA-approved testing.

Pharmacological Action

Gaucher disease is caused by a deficiency of the lysosomal enzyme acid β-glucosidase.

Eliglustat specifically inhibits glucosylceramide synthase (IC50 = 10 ng/mL), reducing the production of GL-1 and thereby alleviating the accumulation of GL-1 in target organs.

By lowering GL-1 production, eliglustat can relieve the accumulation of Gaucher cells in the liver, spleen, and bone marrow, and improve symptoms of organ enlargement and bone disease.

Use Restrictions

CYP2D6 ultrarapid metabolizers may not achieve sufficient eliglustat concentrations to produce therapeutic effects.

For patients with undetermined CYP2D6 genotype (indeterminate metabolizers), a specific dosage cannot be recommended.

Dosage Form, Strength, and Characteristics of Eliglustat (Cerdelga)

Detailed Strength Description

Eliglustat capsules contain eliglustat tartrate, with each capsule containing 84 mg of eliglustat (equivalent to 100 mg of eliglustat tartrate).

Physical Characteristics

The capsules have a pearlescent blue-green opaque cap and a pearlescent white opaque body, with "GZ02" printed in black on the capsule.

Dosage Form Composition

Active ingredient: Eliglustat.

Inactive ingredients: Microcrystalline cellulose, lactose monohydrate, hypromellose, glycerol behenate, gelatin, Candurin Silver Fine Powder, yellow iron oxide, and FD&C Blue No. 2.

Packaging Specifications

NDC 58468-0220-1: Carton containing 4 packs of capsules (56 capsules total). Each pack consists of one blister card with 14 capsules and a cardboard wallet.

NDC 58468-0220-2: Carton containing 1 pack of capsules (14 capsules total). Each pack consists of one blister card with 14 capsules and a cardboard wallet.

Storage Conditions for Eliglustat (Cerdelga)

Storage Conditions

The appropriate storage temperature is 68°F–77°F (20°C–25°C), with brief fluctuations allowed between 59°F and 86°F (15°C and 30°C).

Special Precautions

The medication should be stored in its original packaging.

Keep it out of the reach of children.

Protect from direct sunlight and humid environments.