EU Approves Sanofi’s Orphan Drug CerdelgaBy
Sanofi and its subsidiary Genzyme have been granted marketing authorization by the European Commission (EC) for Cerdelga (eliglustat) capsules, a first-line oral therapy for certain adults living with Gaucher disease Type 1. Cerdelga was approved by the US Food and Drug Administration in August 2014, and the drug is under review by other regulatory authorities.
Sanofi expects that Cerdelga will be available commercially in EU countries beginning in 2015 and over the next few years.
Cerdelga is a potent, highly specific ceramide analogue inhibitor of glucosylceramide synthase with broad tissue distribution including to bone marrow. It reduces the production of glucosylceramide, the substance that builds up in the cells and tissues of people with Gaucher disease Type 1. Cerdelga is indicated in the European Union for the long-term treatment of adult patients with Gaucher disease Type 1 (GD1), who are CYP2D6 poor metabolizers (PMs), intermediate metabolizers, or extensive metabolizers.
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. 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.