It is expected that the European Commission (EC) will make a final decision on granting marketing authorisation for Cerdelga in the European Union (EU) in the coming months. Cerdelga was cleared by the US FDA in August. The therapy is currently under review by other regulatory authorities around the world.
Dr. David Meeker, Genzyme president and CEO, said CHMP's positive opinion is the next step in making Cerdelga available to eligible adults with Gaucher disease type 1 living in the EU. The standard of care for Gaucher disease is Cerezyme (imiglucerase for injection), he noted. With the availability of an effective oral therapy, Cerdelga would give some patients and their physicians more choice in how to manage this disease, Meeker added.
CHMP's opinion was based on results from the Cerdelga clinical development programme, which Genzyme considers to be the largest ever programme carried out in Gaucher disease, involving about 400 patients in 29 countries. Genzyme has been researching an oral therapy for Gaucher disease for fifteen years.
Cerdelga is a novel glucosylceramide analogue given orally. It was designed to partially inhibit the enzyme glucosylceramide synthase, which results in reduced production of glucosylceramide. Glucosylceramide is the substance that builds up in the cells and tissues of people with Gaucher disease.
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