Cerdelga

About Cerdelga:

• Cerdelga contains the active substance Eliglustat which works by blocking the enzyme action involved in producing fat called glucosylceramide. 
• This fat accumulates in the spleen, liver, and bones and is responsible for the symptoms of type-1 Gaucher disease and if the production is reduced, it helps the affected organs to perform better.
• Gaucher disease is a genetic condition and Cerdelga was designated as an ‘orphan medicine’.

Strength: 

Cerdelga is available as 84 mg hard gelatin capsules, with a pearl blue-green opaque cap and pearl white opaque body. Each capsule contains 100 mg of Eliglustat tartrate, which is equivalent to 84 mg of Eliglustat.

Recommended Dosage:

• The recommended dosage of Cerdelga is 84 mg twice daily in CYP2D6 EMs and IMs. 
• The recommended dosage in CYP2D6 PMs is 84 mg once daily and appropriate adverse event monitoring is recommended.
• Swallow capsules whole with water, and do not crush, dissolve, or open the capsule shell.
• Cerdelga may be taken with or without food.
• The consumption of grapefruit with Cerdelga should be avoided as grapefruit is a CYP3A inhibitor.
• If a dose is missed, the prescribed dose should be taken at the next scheduled dose.
• For patients currently on imiglucerase, velaglucerase alfa, or taliglucerase alfa treatment, Cerdelga should be administered 24 hours after the last dose of the previous enzyme replacement therapy.

Warnings & Precautions:

• Peripheral neuropathy: Follow-ups to be done with neurological evaluations at 6-month intervals in such patients.
• Tremor: Dose to be reduced to ameliorate tremor or discontinue treatment if it is not resolved within days of dose reduction.
• Diarrhea and weight loss: Gastrointestinal disease evaluation to be performed in patients who are not showing any response to usual interventions.
• Reductions in Platelet Count: Mild reductions in platelet counts without association with bleeding are observed so monitoring of platelet counts is recommended.

Common Cerdelga Side Effects

• Fatigue
• Headache
• Nausea
• Diarrhea
• Back pain
• Pain in extremities and upper abdominal pain

Use in Specific Population

Pregnancy: Cerdelga has no adequate or well-controlled studies in pregnant women. It is a pregnancy category C drug.

Pediatric use: The safety and effectiveness of pediatric patients have not been established.

Nursing Mothers: It’s unknown if Cerdelga is in human milk. Due to potential adverse reactions in nursing infants, a decision should be made to either discontinue nursing or stop the drug, considering its importance to the lactating woman.

Kidney Impairment: No dosage adjustment is needed for patients with mild renal impairment. Cerdelga has not been studied in patients with moderate to severe renal impairment or end-stage renal disease (ESRD), and its use in these patients is not recommended.  

Liver Impairment: Cerdelga has not undergone studies in patients with hepatic impairment. The use of Cerdelga is not recommended in all stages of hepatic impairment or cirrhosis.

Storage and Handling

• Stored at 20°C to 25°C (68°F to 77°F). 
• Permitted excursions are between 15°C to 30°C (59°F to 86°F).