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Very Rare Disease Treatment Cerdelga® (eliglustat) To Be Made Available On The NHS, Reversing Previous Guidance

Cerdelga is the first oral treatment for adults with type 1 Gaucher disease to be recommended by NICE


Guildford, UK – 31 May, 2017 - Sanofi and its specialty care global business unit, Sanofi Genzyme, announced today that the National Institute of Health and Care Excellence (NICE) has issued a positive Final Evaluation Determination (FED) recommending Cerdelga® (eliglustat) as a first-line treatment for adult patients with type 1 Gaucher disease (the most common form of the disease)1, within its marketing authorisation. This ruling supersedes NICE’s previous provisional negative guidance on eliglustat NHS use2.

"The news that NHS England will fund an oral treatment for type 1 Gaucher disease in England will be welcomed within the Gaucher community," comments Tanya Collin-Histed, Chief Executive, Gauchers Association, UK. “With this decision patients in England, in consultation with their Gaucher Consultant, will be able to choose a treatment that best suits their needs. Having the option of either oral or IV therapy available on the NHS will give many patients access to a new therapeutic opportunity.”

Gaucher disease is a very rare and inherited genetic condition2 which can be life-threatening in severe cases3 . It is incurable,  but can be managed with effective treatment4. The current standard of care in the UK is enzyme replacement therapy (delivered by IV infusion)2. Long-term reliance on IV infusions are not suitable or practical for all. Eliglustat, an oral twice-daily pill, has comparable efficacy and tolerability levels to existing therapy, therefore offering many patients a viable alternative to treatment delivered by IV infusion5.

“Sanofi has been working for over 30 years to help alleviate the lifelong burden that Gaucher disease places on patients and their families. Those living with the condition in England are one step closer to gaining access to Cerdelga, the first NICE-recommended oral alternative to IV infusion therapy,” said Peter Kuiper, General Manager UK & Ireland at Sanofi Genzyme. “Cerdelga is an established first-line treatment in many countries. We hope that other UK national regulatory bodies will follow NICE’s example, allowing patients access to the same choice of treatment as those living in England and elsewhere in the world.”

Eliglustat was designated by the European Commission as an orphan medicine (a medicine used in rare diseases) on 4 December 2007 and was granted a marketing authorisation in the European Union on 19 January 2015. In addition to the EU, eliglustat is approved in 19 countries worldwide and its development programme is the largest-ever in Gaucher disease, with almost 400 patients in 29 countries. Sanofi Genzyme has an ongoing commitment to the advancement of rare disease treatment and is investigating new treatments for patients with rare diseases.

About the NICE process

By publishing a Final Evaluation Determination (FED), NICE has made final recommendations on how eliglustat should be used in the NHS. The FED forms the basis of the final guidance to the NHS in England and, pending no change, is expected in June, 2017. Once the final guidance is published the NHS will be legally obliged to make eliglustat available for routine use within 90 days.

About Gaucher disease

Gaucher disease is a very rare inherited condition which leads to enlargement of the spleen and liver, pain, bone damage and a variety of other uncomfortable symptoms2. It is caused by a deficiency of an enzyme (glucocerebrosidase) which leads to the build-up of complex lipids (fatty substances) in some types of blood cells. These are known as Gaucher cells and occur throughout the liver, spleen, bone marrow and occasionally the lungs2.

All types of Gaucher disease can mimic the signs and symptoms of leukaemia, multiple myeloma and non-Hodgkin’s lymphoma including pain, fatigue, anaemia, jaundice, bone damage, and enlargement of the liver and spleen2,6.

A very rare disease is defined by NICE as a disease with a prevalence of less than 1 in 50,0002. Gaucher disease is more common in people of Ashkenazi family origin, with a frequency of approximately 1 in 500 to 1 in 1000 live births.2 Although there is limited epidemiology data in the UK, it is estimated that over 90% of people affected have type 1 Gaucher disease.2

There are three subtypes of Gaucher disease, of which type 1 is the most prevalent - more than nine out of 10 of people with Gaucher disease in the UK have this type2. Type 2 is the most dangerous form of the disease4, but type 1 disease can also be life-threatening in severe cases3.

About eliglustat

Eliglustat is licensed for the long-term treatment of type 1 Gaucher disease in adults who have the ability to break down (metabolise) certain drugs. A patient’s metaboliser status is determined by a simple blood test which measures how their body breaks down medicine, patients with normal speed (known as CYP2D6 intermediate metabolisers and extensive metabolisers) or slow speed (known as CYP2D6 poor metabolisers) can receive the treatment5. Eliglustat is not recommended for use in CYP2D6 ultra-rapid metabolisers.

Unlike the enzyme replacement therapies, eliglustat is a substrate reduction therapy that partially inhibits an enzyme called glucosylceramide synthase, resulting in reduced production of glucosylceramide (the fatty substance which accumulates in people with Gaucher disease) and Gaucher cells5.

Across two pivotal Phase III studies the efficacy and tolerability of eliglustat has been assessed in adult patients with type 1 Gaucher disease who have had no prior treatment (ENGAGE trial), plus those who have previously been stabilised on enzyme replacement therapy (ENCORE trial)5.

For full eliglustat prescribing information, the Summary of Product Characteristics can be found here: https://ec.europa.eu/health/documents/community-register/2015/20150119130463/anx_130463_en.pdf

About Sanofi

Sanofi, a global healthcare leader, discovers, develops and distributes therapeutic solutions focused on patients' needs. Sanofi is organised into five global business units: Diabetes and Cardiovascular, General Medicines and Emerging Markets, Sanofi Genzyme, Sanofi Pasteur and Consumer Healthcare. Sanofi is listed in Paris (EURONEXT: SAN) and in New York (NYSE: SNY).

Sanofi Genzyme focuses on developing specialty treatments for debilitating diseases that are often difficult to diagnose and treat, providing hope to patients and their families.

The black triangle means this medicine is subject to additional monitoring. This will allow quick identification of new safety information. Side effects of medicines should be reported to your doctor or pharmacist.


[1] The National Institute for Health and Care Excellence. Available at: https://www.nice.org.uk/guidance/indevelopment/gid-gaucherdiseasetype1eliglustatid709.  Last accessed May 2017

[2] The National Institute for Health and Care Excellence. Available at: : https://www.nice.org.uk/guidance/GID-GAUCHERDISEASETYPE1ELIGLUSTATID709/documents/evaluation-consultation-document. Last accessed May 2017

[3] Gaucher Association. Type 1. Available at: https://www.gaucher.org.uk/about_gaucher/type_1. Last accessed May 2017

[4] Gauchers Association. About Gaucher. Available at: https://www.gaucher.org.uk/about_gaucher/type_2. Last accessed May 2017

[5] Eligulstat EU SPC. Available at: http://ec.europa.eu/health/documents/community-register/2015/20150119130463/anx_130463_en.pdf/. Last accessed May 2017

[6] Mistry PK. Consequences of diagnostic delays in type 1 Gaucher disease: the need for greater awareness among Hematologists-Oncologists and an opportunity for early diagnosis and intervention. Am J Hematol. 2007;82(8):697-701

[7] Clinical Recommendation Committee. 2010. Available at: https://www.gov.im/media/631127/orphanorultraorphandruginterven.pdf. Last accessed May 2017

Date of preparation: May 2017 - Job bag number: GZUK.CERD.17.05.0253

Updated: May 31, 2017

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