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Rare diseases - Cerdelga, a drug for Gaucher disease: important safety information


Cerdelga ( Eliglustat ), a novel glucosylceramide analog given orally, was designed to partially inhibit the enzyme glucosylceramide synthase, which results in reduced production of glucosylceramide. Glucosylceramide is the substance that builds up in the cells and tissues of people with Gaucher disease.

Following an extensive pre-clinical and early clinical research program, Cerdelga was studied in the largest phase 3 clinical program ever conducted in Gaucher disease.
Cerdelga capsules are indicated for the long-term treatment of adults 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.
Patients who are CYP2D6 ultra-rapid metabolizers ( URMs ) may not achieve adequate concentrations of Cerdelga to achieve a therapeutic effect.
A specific dose cannot be recommended for those patients whose CYP2D6 genotype cannot be determined ( indeterminate metabolizers ).

Important safety information

Cerdelga is contraindicated in the following patients due to the risk of significantly increased plasma concentrations which may result in prolongation of the PR, QTc, and/or QRS cardiac intervals that could result in cardiac arrhythmias: EMs or IMs taking a strong or moderate CYP2D6 inhibitor concomitantly with a strong or moderate CYP3A inhibitor and IMs or PMs taking a strong CYP3A inhibitor.
Drugs that inhibit CYP2D6 and CYP3A may significantly increase the exposure to Cerdelga; Cerdelga dose adjustment may be needed, depending on metabolizer status.

Because Cerdelga is predicted to cause increases in ECG intervals at substantially elevated plasma concentrations, use is not recommended in patients with pre-existing cardiac disease, long QT syndrome, or in combination with Class IA and Class III antiarrhythmic medications.

The most common adverse reactions ( greater than or equal to 10% ) for Cerdelga are: fatigue, headache, nausea, diarrhea, back pain, pain in extremities, and upper abdominal pain.

Only administer Cerdelga during pregnancy if the potential benefit justifies the potential risk; based on animal data, Cerdelga may cause fetal harm. Discontinue drug or nursing based on importance of drug to mother.

Cerdelga is not recommended in patients with moderate to severe renal impairment or in patients with hepatic impairment. ( Xagena )

Source: Genzyme, 2014

XagenaMedicine_2014



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