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Confidence in Cerdelga® (eliglustat) - The ONLY first-line oral therapy for adults with Gaucher disease type 1 with long-term efficacy data in treatment-naïve patients and patients previously stabilised on enzyme replacement therapy1

Efficacy icon

Cerdelga® demonstrated comparable efficacy to enzyme replacement therapy (ERT) in patients previously stabilised on ERT through maintenance of patient stability across haematological and visceral parameters1

Clinical trials icon

In the clinical trials, the proportion of patients who confirmed preference for an oral treatment was 94% (93/99)2


Cerdelga® demonstrated efficacy vs placebo in visceral and haematological parameters1

In treatment-naïve patients, Cerdelga® demonstrated efficacy vs placebo in visceral and haematologic parameters over 9 months, with continued improvements in Cerdelga®-treated patients for up to 4.5 years.*1

Cerdelga® demonstrated comparable efficacy to imiglucerase in maintenance of patients’ stability across visceral and haematologic parameters in patients previously stabilized on ERT.**1 The stability in visceral and haematologic parameters was maintained for up to 4 years.†1

Primary Composite Endpoint

Stability in 4 parameters; haemoglobin levels, platelet count, spleen and liver volume

primary composite endpoint

In an open-label, phase 2 study, Cerdelga® showed sustained improvements in organ volume and haematologic parameters over an 8-year treatment period in treatment-naïve patients (n=18).‡1

*All patients transitioned to Cerdelga® treatment after 9 months.
**Excludes patients with a total splenectomy and all patients transitioned to Cerdelga® after 52 weeks.
†Excludes patients with a total splenectomy.
‡18 patients completed 8 years of treatment. 16 patients had an efficacy endpoint assessment at year 8.

Cerdelga® improved skeletal measures in treatment-naïve patients and patients previously stabilised on ERT1

Skeletal measures (bone marrow burden (BMB) and bone mineral density (BMD) scores) improved during Cerdelga® treatment in treatment-naïve patients. BMD remained stable under Cerdelga® treatment in patients previously stabilised on ERT.1

Cerdelga® improved BMD from osteopenia to normal range after 4 years3

Cerdelga improvment data


Cerdelga® is generally of manageable tolerability1

  • No adverse events have been reported as ‘very common’ (>10%)1
  • The most commonly reported AE with Cerdelga® is dyspepsia, in approximately 6% of the clinical trial patients1
Adverse reactions1
System organ class
Common (≥1/100 to <1/10)
Nervous System disorders Headache, dizziness, dysgeusia
Cardiac disorders Palpitations
Respiratory, thoracic and mediastinal disorders Throat irritation
Gastrointestinal disorders Dyspepsia, abdominal pain upper, diarrhoea, nausea, constipation, abdominal pain, gastroesophageal reflux disease, abdominal distension, gastritis, dysphagia, vomiting, dry mouth, flatulence
Skin and subcutaneous tissue disorders Dry skin, urticaria
Musculoskeletal and connective tissue disorders Arthralgia, pain in extremity, back pain
General disorders and administration site conditions Fatigue

The overall adverse reaction profile of Cerdelga® is based on a total of 393 patients from four studies between the ages of 16-75 years who received eliglustat for a median duration of 3.5 years (up to 9.3 years). This includes the pooled results from the primary analysis periods and extension periods of two pivotal Phase 3 studies (ENGAGE and ENCORE), one 8-year, long term Phase 2 study (Study 304) and one supporting Phase 3b study (EDGE).

Cerdelga® (eliglustat) drug-drug interaction checker

Cerdelga® is contraindicated in patients who are CYP2D6 intermediate metabolisers (IMs) or extensive metabolisers (EMs) taking a strong or moderate CYP2D6 inhibitor concomitantly with a strong or moderate CYP3A inhibitor, and in patients who are CYP2D6 poor metabolisers (PMs) taking a strong CYP3A inhibitor. Under these conditions both major metabolic pathways for eliglustat metabolism are impaired, with predicted substantially elevated eliglustat plasma concentration. Cerdelga® is contraindicated in CYP2D6 extensive metabolisers with severe hepatic impairment. Cerdelga® is contraindicated in CYP2D6 extensive metabolisers with mild or moderate hepatic impairment taking a strong or moderate CYP2D6 inhibitor. Please note that this tool is only designed to give guidance on the effect of plasma concentrations of Cerdelga® when used in combined with other medications and may not account for other interactions.

To access the drug-drug interaction checker click here

The patient’s experience

After 1 year on Cerdelga®, 98% (138/141) of patients with Gaucher who switched to Cerdelga® after long-term ERT preferred oral treatment over intravenous therapy.4

To further understand patients’ treatment decisions and their experience with Cerdelga®, watch our videos based on real patients’ stories.

Patient videos







Managing Gaucher disease is a lifelong commitment, and we understand that each patient has unique needs.

We have designed the Cerdelga® support programme to aid in your discussions about treatment with patients and to provide them with ongoing support with Gaucher disease whilst on Cerdelga®.

To access post–prescription patient materials that can be ordered or downloaded, click here

Cerdelga® mechanism of action

Cerdelga® is a potent and specific inhibitor of glucosylceramide synthase. Cerdelga® is a substrate reduction therapy (SRT) with clinical studies showing wide distribution to tissues, including bone marrow.1

Cerdelga® reduces the rate of synthesis of the major substrate glucosylceramide (GL-1) to match its impaired rate of catabolism in patients with Gaucher disease Type 1.1

Cerdelga® is a ceramide-based analogue (versus amino-sugar-based analogue). It is a specific inhibitor of glucosylceramide synthase5–7 and does not inhibit intestinal glucosidases.5,6

Please view the video of Cerdelga® mode of action, below:

CYP2D6 testing

Cerdelga® should only be used in patients who, based on genotyping, have a predicted CYP2D6 poor, intermediate or extensive metaboliser phenotype. Determination of the patient's CYP2D6 phenotype is required prior to initiating treatment with Cerdelga®.1

Genotyping to determine the patient's CYP2D6 phenotype is to be performed using an established genetic laboratory test that is able to detect a specific set of CYP2D6 allelles with adequate accuracy, sensitivity and specificity in order to ensure consistent identification of CYP2D6 metaboliser status.

Sanofi Genzyme offers a CYP2D6 testing service to UK clinicians treating patients with Gaucher disease through LabCorp Ltd. However, there are several suitable commercial tests available.

In the UK, for more information on gaining access to testing via LabCorp Ltd. or for accredited laboratories, you can contact Sanofi Genzyme on 0800 035 2525, or your local representative.

Useful links


DDI Tool

This tool has been designed to help quickly navigate through the drug-drug interactions (DDIs) for Cerdelga® as outlined in the SmPC based on the metabolizer status of a patient.
Prescribing decisions should be made in consultation with an expert in the management of patients with Gaucher disease.



UK-based charity dedicated to raising awareness of Gaucher disease, and those affected and their families. Provides support and information for those affected by Gaucher disease.



To support you in identifying and treating your patients with Gaucher disease, Sanofi Genzyme have launched a free access e-learning module: Identifying and Treating Gaucher disease.


  1. Cerdelga®. Summary of Product Characteristics.
  2. Cox TM et al. Lancet. 2015 Jun 13;385(9985):2355–62.
  3. Lukina E et al. Blood Cells Mol Dis. 2014 Dec;53(4):274–6.
  4. Cox TM et al. Blood. 2017 Apr 27;129(17):2375–2383.
  5. Belmatoug N et al. Eur J Intern Med. 2016. pii: S0953–6205(16)30217–5.
  6. McEachern KA et al. Mol Genet Metab. 2007;91:259–267
  7. Shayman JA. Drugs Fut. 2010;35:613–620.