EU approves Cerdelga (eliglustat) for treatment of Gaucher disease type 1-Sanofi/Genzyme

Sanofi and its subsidiary Genzyme announced that the European Commission (EC) has granted marketing authorization for Cerdelga (eliglustat) capsules, a first line oral therapy for certain adults living with Gaucher disease type 1. A small number of adult patients who metabolize Cerdelga more quickly or at an undetermined rate, as detected by an established genetic laboratory test, will not be eligible for Cerdelga treatment. Cerdelga was approved by the FDA in August 2014, and is under review by other regulatory authorities around the world. It is expected that Cerdelga will be available commercially in EU countries beginning in 2015 and over the next few years.

Comment: Non-neuropathic type 1 Gaucher disease is an autosomal recessive inherited disease caused by the deficiency or absence of beta glucocerebrosidase (beta glucosidase). The highest prevalence of type 1 is in Ashkenazi Jews, but it affects all ethnic groups. It manifests at any age but is seen predominantly in the first two decades.

Other drugs available for Gaucher Disease are Vpriv (velaglucerase alfa) and Elelyso (taliglucerase alfa) and these are both injected. Zavesca (miglustat) is an oral drug for type I Gaucher Disease but this is only used in people who cannot be treated with enzyme replacement therapy.