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European CHMP Adopts Positive Opinion for Genzyme's Cerdelga (eliglustat) Capsules
2014-11-23 22:11:47

21.November 2014
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Genzyme, a Sanofi company, announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending approval of Cerdelga® (eliglustat) capsules, an oral treatment for certain adults living with Gaucher disease type 1.

The European Commission (EC) is expected to make a final decision on granting marketing authorization for Cerdelga in the EU in the coming months. Cerdelga was approved by the U.S. Food and Drug Administration in August, and is under review by other regulatory authorities around the world.

“Today’s CHMP opinion is the next step in ensuring that Cerdelga is available to eligible adults with Gaucher disease type 1 living in the European Union,” said David Meeker, MD, Genzyme President and CEO. “The standard of care for Gaucher disease is Cerezyme® (imiglucerase for injection). With the availability of an effective oral therapy, Cerdelga would offer some patients and their physicians more choice in how to manage this serious disease.”

The CHMP opinion was based on data from the Cerdelga clinical development program, which is the largest ever conducted in Gaucher disease, with approximately 400 patients treated in 29 countries. Genzyme has been researching an oral therapy for Gaucher disease for fifteen years.

The most common adverse reactions (greater than or equal to 10%) are fatigue, headache, nausea, diarrhea, back pain, pain in extremities, and upper abdominal pain.