NICE U-turns to recommend Sanofi Gaucher disease drug
pharmafile | June 2, 2017 | News story | Medical Communications, Sales and Marketing | Gaucher disease, NICE, Sanofi
It has been revealed that NICE has reached the decision to recommend Sanofi’s Cerdelga (eligustat) for use on the NHS in England and Wales as a first-line treatment of type 1 Gaucher disease in adult patients, reversing a previous decision rejecting its use.
A rare, inherited genetic condition, Gaucher disease is characterised by the enlargement of the spleen and liver as well as pain and bone damage. The illness is caused by cells – known as Gaucher cells – which populate the liver, spleen, bone marrow and lungs and originate from complex lipid build-up in the blood cells as a result of glucocerebrosidase deficiency. It affects less than one in 50,000, with type 1 being the most common variety, accounting for 90% of cases.
There is no known cure for the sometimes life-threatening disease, though it can be managed through treatment, namely glucocerebrosidase replacement via intravenous infusion. Cerdelga offers a much more practical treatment solution, administered as an oral twice-daily pill.
“Sanofi has been working for over 30 years to help alleviate the lifelong burden that Gaucher disease places on patients and their families,” commented Peter Kuiper, General Manager, UK & Ireland at Sanofi Genzyme. “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. 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.”
Matt Fellows
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