Sanofi Disappointed by Provisional NICE Decision Not To
Approve Very Rare Disease Treatment Cerdelga® ▼ (eliglustat)
For Use in the NHS
- Eliglustat not recommended as a treatment option for adults with type 1
Gaucher disease in England, according to draft guidance
Sanofi and its specialty care global business unit, Sanofi Genzyme, are disappointed about the
provisional recommendation by the National Institute of Health and Care Excellence (NICE) to not
recommend eliglustat as a first-line oral treatment for adults with type 1 Gaucher disease in England.
NICE published their draft guidance today in an Evaluation Consultation Document (ECD)1; this is
not yet final guidance and may be subject to change.
“We are taking immediate action to better understand NICE’s position and the issues that led to this
provisional recommendation.” said Peter Kuiper, General Manager UK & Ireland at Sanofi
Genzyme. “Sanofi has gone to considerable effort to ensure this medicine benefits both patients
and the NHS. Whilst today’s announcement is a disappointment, we are committed to working
closely with NICE and the Gaucher community to allow eliglustat to become routinely available on
the NHS for patients in England”.
Peter Kuiper added: “Although patients currently have access to existing therapies for the treatment
of Gaucher disease, which have been supported by NHS commissioning for many years, eliglustat
represents another step forward. At Sanofi Genzyme, we have been dedicated for more than 30
years to advancing the science behind Gaucher disease treatments and will in response to this
ECD again submit our argument on why we believe this medicine represents good value for the
NHS, based on both, how eliglustat works compared with existing treatments and the cost of
treatment.”
As part of the HST submission to NICE, Sanofi proposed a simple patient access scheme that was
accepted by the Department of Health. Patient access schemes allow manufacturers to subsidise
the cost or discount the price the NHS pays for innovative medicines.
This is the first time NICE has evaluated the benefits and costs of using eliglustat within its licensed
indication. Sanofi Genzyme will respond to publication of this ECD as part of the ongoing official
consultation process.
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.
For full prescribing information about eliglustat, the Summary of Product Characteristics can be
found here: https://ec.europa.eu/health/documents/communityregister/2015/20150119130463/anx_130463_en.pdf
▼ 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.
GZUK.CERD.17.03.0123
Date of preparation: March 2017
References
1
The National Institute for Health and Care Excellence. Available at: https://www.nice.org.uk/guidance/indevelopment/gidgaucherdiseasetype1eliglustatid709 . Last accessed March 2017