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

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

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 parameters.1

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

  • Stability in 4 parameters; haemoglobin levels, platelet count, spleen and liver volume.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® 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 patients.1

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 incidence of the adverse reaction was the same or higher with placebo than with Cerdelga in the placebo-controlled pivotal study.

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).

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.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.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


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 alleles with adequate accuracy, sensitivity and specificity in order to ensure consistent identification of CYP2D6 metaboliser status.

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

In the UK, for more information on gaining access to testing via Archimed Life, or for accredited laboratories, you can contact Sanofi Medical Information on 0800 035 2525, or your local representative.

Product Inspiration

Patient story Miranda

Patient story Rob

Patient story Mike

    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.

MAT-XU-2201107 (v2.0)
Date of preparation: January 2023