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Very rare disease treatment Cerdelga® (eliglustat) will be made available in Scotland for Gaucher patients

• The oral pill offers Gaucher patients and their physicians an alternative to current standard of care delivered by IV infusion
• Gaucher disease is an inherited, incurable condition that can lead to enlargement of the spleen and liver, pain, bone damage among other symptoms
• The positive decision means NHS Scotland is aligned with NHS England in funding eliglustat, reducing potential disparities in care


Guildford, UK – 11 December 2017– Sanofi and its specialty care global business unit, Sanofi Genzyme, announced today that the Scottish Medicines Consortium (SMC) has accepted 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.

NHS Scotland is now aligned with NHS England in funding eliglustat, thereby reducing potential disparities in care across the UK for patients with the rare inherited condition.2

“The Gaucher community is pleased that NHS Scotland will fund an oral treatment for adults living with type 1 Gaucher disease,” says Tanya Collin-Histed, Chief Executive, Gauchers Association, UK. “With this decision, Scottish Gaucher patients and their physicians have the ability to choose between an oral or IV treatment based on patients’ individual needs.”

Gaucher disease is a very rare and inherited genetic condition,2 which can be life-threatening in severe cases.3 It is incurable, but can be managed with effective treatment.4 The current standard of care in the UK is enzyme replacement therapy (ERT), which is delivered by IV infusion.2Long-term reliance on IV infusions are not suitable or practical for all.

Decision allows access to oral pill alternative to IV infusion

Eliglustat, an oral twice-daily pill, has comparable efficacy and tolerability levels to existing therapy, therefore offering Gaucher patients a viable alternative to treatment delivered by IV infusion.1

“We have been dedicated to working productively with the SMC to ensure Gaucher patients have access to Cerdelga on the NHS Scotland,” said Peter Kuiper, General Manager UK & Ireland at Sanofi Genzyme. “This decision echoes the positive decision by NICE earlier this year, meaning Gaucher patients in Scotland and England have the same choice of treatment options.”

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 has been approved in more than 50 countries worldwide, and was studied in the largest-ever clinical development programme for type 1 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 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 symptoms.2 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 lungs.2

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 spleen.2,3

A very rare disease, such as Gaucher disease, is defined by NICE as a disease with a prevalence of less than 1 in 50,000.3 Although Gaucher disease is very rare in the UK population as a whole, it is more common in people of Ashkenazi family origin, with a frequency of 1-2 per 1000 live births.2

There are three subtypes of Gaucher disease, of which type 1 is the most prevalent.2

About eliglustat

Eliglustat is licensed for the long-term treatment of type 1 Gaucher disease in adults but is only allowed to be used in people who have the ability to metabolise (break down) specific types of drugs slowly. 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 treatment.5 Eliglustat is not recommended for use in CYP2D6 ultra-rapid metabolisers.

Unlike 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 cells.5

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: www.medicines.org.uk/emc/medicine/33365

About Sanofi

Sanofi is dedicated to supporting people through their health challenges. We are a global biopharmaceutical company focused on human health. We prevent illness with vaccines, provide innovative treatments to fight pain and ease suffering. We stand by the few who suffer from rare diseases and the millions with long-term chronic conditions.

With more than 100,000 people in 100 countries, Sanofi is transforming scientific innovation into healthcare solutions around the globe.

Sanofi, Empowering Life

For more information please visit www.sanofi.co.uk or on Twitter at @SanofiUK


[1] Scottish Medicines Consortium. SMC Advice . Eliglustat (Cerdelga). Available at: https://www.scottishmedicines.org.uk/SMC_Advice/Advice/1277_17_eliglustat_Cerdelga/eliglustat_Cerdelga. [Last accessed: December 2017]

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

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

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

[5] Eligulstat EU SPC. Available at: www.medicines.org.uk/emc/medicine/33365. [Last accessed: December 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 December 2017]

Date of preparation: December 2017 - Job bag number: GZUK.CERD.17.12.0587

Updated: December 11, 2017

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