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news.The European Commission (EC) has granted marketing authorization for Genzyme's Cerdelga (eliglustat) capsules, a first line oral therapy for certain adults living with Gaucher disease type 1.
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The company said that a small number of adult patients who metabolize Cerdelga more quickly or at an undetermined rate, will not be eligible for Cerdelga treatment.
In August 2014, the company secured approval from the US Food and Drug Administration (FDA) for Cerdelga and it is now under review by other regulatory authorities across the world.
Cerdelga, a potent, highly specific ceramide analogue inhibitor of glucosylceramide synthase, is expected to be commercially available in EU countries beginning in 2015 and over the next few years.
The drug works by reducing the production of glucosylceramide, the substance that builds up in the cells and tissues of people with Gaucher disease type 1.
In the EU, Cerdelga is indicated for the long-term treatment of adult patients with Gaucher disease type 1 (GD1), who are CYP2D6 poor metabolizers (PMs), intermediate metabolizers (IMs) or extensive metabolizers (EMs).
The European approval was based on data from the Cerdelga clinical development program, which included two pivotal Phase III clinical trials involving 400 patients in 29 countries.
In a Phase III placebo-controlled trial (ENGAGE) improvements were observed across the following endpoints after nine months on Cerdelga: spleen size, platelet levels, hemoglobin levels, and liver volume.
The second Phase III trial was designed to evaluate disease stability in patients previously treated with enzyme replacement therapy (ENCORE).