Patients with Gaucher disease in Europe will welcome news this morning that regulators have given marketing clearance to Genzyme’s Cerdelga (eliglustat), offering an oral first-line treatment for the rare genetic disorder.

However, in line with its US approval last August, a small number of adult patients who metabolise Cerdelga more quickly or at an undetermined rate, as detected by an established genetic laboratory test, will not be eligible for treatment, the European Commission said.

Approval came on the back of data from the largest clinical research program ever conducted in Gaucher disease type 1, involving around 400 patients, which showed that Cerdelga improved a variety of endpoints including spleen size, platelet levels, hemoglobin levels, and liver volume, and also showed non-inferiority to the enzyme replacement therapy Cerezyme (imiglucerase). 

On the safety side, the majority of side effect were found to be mild and transient, the Sanofi company said, with the commonly reported being diarrhoea in around 6% of the patients (though the incidence was the same or higher with placebo the study). 

Cerdelga will be available commercially in EU countries beginning in 2015 and over the next few years, the firm noted.