Sanofi's 'disappointment' at NICE decision not to approve rare disease treatment Cerdelga for use in NHS

Amy Schofield 15 March 2017

Sanofi and its specialty care global business unit, Sanofi Genzyme, have expressed their disappointed at 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NICE published its draft guidance in an Evaluation Consultation Document (ECD), however this is not yet final guidance and may be subject to change.

As part of its submission to NICE, Sanofi proposed a simple patient access scheme that was accepted by the Department of Health. These types of schemes allow manufacturers to subsidise the cost, or discount the price that the NHS pays for innovative medicines.

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 EuropeanUnion on 19 January 2015.

This is the first time that 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.

Peter Kuiper, General Manager UK & Ireland at Sanofi Genzyme, said: “We are taking immediate action to better understand NICE’s position and the issues that led to this provisional recommendation. 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”.


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