Sanofi Receives FDA Approval for Gaucher Disease Drug Cerdelga
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The US Food and Drug Administration (FDA) has approved Sanofi’s Cerdelga (eliglustat), a first-line oral therapy for the long-term treatment of certain adult patients with the Type 1 form of Gaucher disease, a rare genetic disorder. The drug received orphan drug status from the FDA.

The new oral drug was developed by Sanofi’s Genzyme subsidiary, which also developed Cerezyme (imiglucerase for injection), which is indicated for long-term enzyme replacement therapy for pediatric and adult patients with a confirmed diagnosis of Type 1 Gaucher disease. Gaucher disease occurs in people who do not produce enough of an enzyme called glucocerebrosidase. The enzyme deficiency causes fatty materials to collect in the spleen, liver, and bone marrow. In patients with Gaucher disease Type 1, Cerdelga slows down the production of the fatty materials by inhibiting the metabolic process that forms them.

Cerdelga is a hard gelatin capsule containing eliglustat that is taken orally. Cerezyme is administered by intravenous infusion over one or two hours. 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 is expected to be available to patients within a month.

Cerezyme posted 2013 sales of EUR 688 million ($913 million) and is one of several enzyme-replacement therapies by Sanofi to treat rare diseases. Others are: Myozyme/Lumizyme (alglucosidase alfa) to treat Pompe disease; Fabrazyme (agalsidase beta) to treat Fabry disease; and  Aldurazyme (laronidase) to treat mucopolysaccharidosis Type I.

Marketing applications for Cerdelga are under review by the European Medicines Agency and other regulatory authorities.

Source: FDA and Sanofi

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