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Home Industry News Genzyme’s Gaucher disease drug wins EU approval

Genzyme’s Gaucher disease drug wins EU approval

22nd January 2015

Genzyme has announced the approval of Cerdelga, an oral first-line treatment for adults with Gaucher disease type 1, by EU regulators.

The drug, generic name eliglustat, was approved for sale in the US in August following the biggest-ever clinical research programme into the efficacy of a treatment pathway for the rare genetic disorder, PharmaTimes reports.

Comprising 400 patients, the study found that Cerdelga contributed to a wide range of positive outcomes, including improved spleen size, platelet levels, hemoglobin levels and liver volume.

It also demonstrated non-inferiority to the enzyme replacement therapy Cerezyme, generic name imiglucerase.

Side effects were shown to be mild, with diarrhoea exhibited in around six percent of patients. A small number of individuals metabolised the drug at an above-average or undetermined rate, prompting regulators to rule that those individuals are ineligible for treatment.

Cerdelga will be rolled out in EU countries from 2015 onwards, Genzyme said in a statement.ADNFCR-8000103-ID-801771411-ADNFCR

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