Eliglustat (Cerdelga) is an oral medication used for the treatment of Type 1 Gaucher Disease (GD1). It alleviates the symptoms of Gaucher Disease by reducing the production of glucosylceramide (GL-1).

Indications for Eliglustat (Cerdelga)

Primary Indication

Eliglustat is indicated for the long-term treatment of adult patients with Type 1 Gaucher Disease, but it must meet specific metabolic status requirements.

Target Population

Patients confirmed by FDA-approved testing to be extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs) of CYP2D6.

Prior to treatment, genetic testing is required to determine the patient's metabolic type, which helps ensure the effectiveness of the medication.

Use Restrictions

Ultra-rapid metabolizers (URMs): These patients may fail to achieve effective plasma drug concentrations, so the use of Eliglustat is not recommended.

Patients with unknown metabolic type: Specific dosage cannot be recommended.

Precautions

For female patients with Type 1 Gaucher Disease, if symptoms are not controlled before and during pregnancy, the risk of spontaneous abortion may increase.

The manifestations of the disease may worsen during pregnancy, requiring close monitoring.

Dosage Form, Strength and Characteristics of Eliglustat (Cerdelga)

Dosage Form and Strength

Capsules, each containing 84 mg of eliglustat.

Appearance Characteristics

The capsule consists of a pearlescent blue-green opaque cap and a pearlescent white opaque body, imprinted with the black marking "GZ02".

Composition

Active Ingredient: Eliglustat tartrate.

Excipients: Microcrystalline cellulose, lactose monohydrate, hypromellose, glyceryl behenate, gelatin, silver lustre pigment, yellow iron oxide, and FD&C Blue No. 2.

Pharmacological Action

By inhibiting glucosylceramide synthase, Eliglustat reduces the accumulation of GL-1 in macrophages, thereby improving symptoms such as hepatosplenomegaly, anemia, and thrombocytopenia.

Storage Method for Eliglustat (Cerdelga)

Temperature Requirement

Store at 20°C to 25°C (68°F to 77°F); short-term storage at 15°C to 30°C (59°F to 86°F) is permitted.

Usage Precautions

Keep the capsules intact; protect from moisture and direct sunlight.

Store out of the reach of children.