Source: European Medicines Agency (EU) Revision Year: 2025 Publisher: Genzyme Europe B.V., Paasheuvelweg 25, 1105 BP Amsterdam, The Netherlands
Cerdelga is indicated for the long-term treatment of adult patients with Gaucher disease type 1 (GD1), who are CYP2D6 poor metabolisers (PMs), intermediate metabolisers (IMs) or extensive metabolisers (EMs).
Cerdelga is indicated for paediatric patients with GD1 who are 6 years and older with a minimum body weight of 15 kg, who are stable on enzyme replacement therapy (ERT), and who are CYP2D6 PMs, IMs or EMs.
Therapy with Cerdelga should be initiated and supervised by a physician knowledgeable in the management of Gaucher disease.
Before initiation of treatment with Cerdelga, patients must be genotyped for CYP2D6 to determine the CYP2D6 metaboliser status.
Eliglustat should not be used in patients who are CYP2D6 ultra-rapid metabolisers (URMs) or indeterminate metabolisers (see section 4.4).
The recommended dose is 84 mg eliglustat twice daily in CYP2D6 IMs and EMs.
The recommended dose is 84 mg eliglustat once daily in CYP2D6 PMs.
Table 1. Paediatric population (from 6 to <18 years of age) weighing ≥15 kg:
| Weight | CYP2D6 EMs and IMs | CYP2D6 PMs |
|---|---|---|
| ≥50 kg | 84 mg twice daily | 84 mg once daily |
| 25 to <50 kg | 84 mg twice daily | 42 mg once daily |
| 15 to <25 kg | 42 mg twice daily | 21 mg once daily |
Cerdelga is to be taken orally in children who can swallow intact capsule.
If a dose is missed, the prescribed dose should be taken at the next scheduled time; the next dose should not be doubled.
There is limited experience in the treatment of elderly with eliglustat. Data indicates that no dose adjustment is considered necessary (see sections 5.1 and 5.2).
Table 2. Patients with hepatic impairment:
| CYP2D6 metaboliser type | Hepatic Impairment | Inhibitors | Dose adjustment |
|---|---|---|---|
| EM | Mild (Child-Pugh class A) | Eliglustat alone | No dose adjustment required |
| Moderate (Child-Pugh class B) | Eliglustat alone | Not recommended (see section 5.2) | |
| Severe (Child-Pugh class C) | Eliglustat alone Eliglustat + Any CYP inhibitor | Contraindicated (see sections 4.3 and 5.2) | |
| Mild (Child-Pugh class A) or moderate (Child-Pugh class B) | Eliglustat + strong or moderate inhibitor of CYP2D6 | Contraindicated (see sections 4.3 and 5.2) | |
| Mild (Child-Pugh class A) | Eliglustat + weak inhibitor of CYP2D6; or strong, moderate or weak inhibitor CYP3A | Once daily dose should be considered (see sections 4.4 and 5.2) | |
| IM or PM | Any | N/A | Not recommended (see section 5.2) |
Table 3. Patients with renal impairment:
| CYP2D6 metaboliser type | Renal impairment | Dose adjustment |
|---|---|---|
| EM | Mild, moderate or severe | No dose adjustment required (see sections 4.4 and 5.2) |
| End stage renal disease (ESRD) | Not recommended (see sections 4.4 and 5.2) | |
| IM or PM | Mild, moderate or severe, or ESRD | Not recommended (see sections 4.4 and 5.2) |
Safety and efficacy data of eliglustat are limited in paediatric patients below the age of 6 years. There are no data to support the use of eliglustat in children weighing less than 15 kg. Currently available data are described in section 5.1.
Cerdelga is to be taken orally. The capsules should be swallowed whole, preferably with water, and must not be crushed or dissolved.
The capsules may be taken with or without food. Consumption of grapefruit or its juice should be avoided (see section 4.5).
Mixing the content of the capsule (eliglustat powder) into food or drinks has not been studied.
The highest eliglustat plasma concentration observed to date occurred in a Phase 1 single-dose dose escalation study in healthy subjects, in a subject taking a dose equivalent to approximately 21 times the recommended dose for GD1 patients. At the time of the highest plasma concentration (59-fold higher than normal therapeutic conditions), the subject experienced dizziness marked by disequilibrium, hypotension, bradycardia, nausea, and vomiting.
In the event of acute overdose, the patient should be carefully observed and given symptomatic treatment and supportive care.
3 years.
This medicinal product does not require any special storage conditions.
PETG/COC.PETG/PCTFE-aluminium blister.
Each blister wallets contains 14 hard capsules.
Each pack contains 14, 56 or 196 hard capsules.
Not all pack sizes may be marketed.
Any unused product or waste material should be disposed of in accordance with local requirements.
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