Sanofi subsidiary Genzyme has received marketing approval from European Commission (EC) for its Cerdelga (eliglustat) capsules to treat adult patients with Gaucher disease type 1.
Approval was granted for first line oral therapy to treat adult patients with Gaucher disease type 1 (GD1), who are CYP2D6 poor metabolisers (PMs), intermediate metabolisers (IMs) or extensive metabolisers (EMs).
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Cerdelga is a specific ceramide analogue inhibitor of glucosylceramide synthase, with broad tissue distribution including to bone marrow.
It helps in reducing production of glucosylceramide, which builds up in the cells and tissues of people with Gaucher disease type 1.
Genzyme noted that 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 Cerdelga treatment.
Approval was based on data from the Cerdelga clinical development programme, which included 400 patients treated in 29 countries. The programme comprised two pivotal Phase III clinical trials.
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By GlobalDataIn a Phase III placebo-controlled trial, improvements were observed at end-points, including spleen size, platelet levels, hemoglobin levels, and liver volume after nine months, noted Genzyme.
According to the company, the second Phase III trial met pre-specified criteria for non-inferiority to an enzyme replacement therapy, which was a composite end-point of each of the following parameters, including spleen volume, hemoglobin levels, platelet counts, and liver volume.
Genzyme plans to commercialise the drug in EU countries beginning in 2015, and over the next few years. Cerdelga received approval from US Food and Drug Administration in August 2014.
It is under review by other regulatory authorities worldwide, noted Genzyme. Gaucher disease is an inherited condition, which affects fewer than 10,000 people worldwide.
Image: Very high magnification micrograph of Gaucher disease and necrotic bone. Photo: courtesy of Nephron.
