NICE backs new drug for Gaucher disease

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, a very rare and inherited genetic condition which can be life-threatening in severe cases.

This means that Cerdelga, from French pharma major Sanofi (Euronext: SAN) and its specialty care global business unit Sanofi Genzyme, is the first oral treatment for many adults with the condition to be recommended by the NICE, the cost-effectiveness watchdog for England and Wales.

The current standard of care in the UK is enzyme replacement therapy (delivered by IV infusion). Long-term reliance on IV infusions is 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 treat ment delivered by IV infusion. This ruling supersedes the NICE’s previous provisional negative guidance on eliglustat National Health Service use, Sanofi noted.