European CHMP Adopts Positive Opinion for Genzyme’s Cerdelga® (eliglustat) Capsules

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 treating 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.

About Gaucher Disease

Gaucher disease is an inherited condition affecting fewer than 10,000 people worldwide. People with Gaucher disease do not have enough of an enzyme, β-glucosidase (glucocerebrosidase) that breaks down a certain type of fat molecule. As a result, lipid engorged cells (called Gaucher cells) amass in different parts of the body, primarily the spleen, liver and bone marrow. Accumulation of Gaucher cells may cause spleen and liver enlargement, anemia, excessive bleeding and bruising, bone disease and a number of other signs and symptoms. The most common form of Gaucher disease, type 1, generally does not affect the brain.

About Genzyme’s Gaucher Disease Program

Driven by a long-term commitment to meeting unmet needs in the Gaucher community, Genzyme has been researching and developing treatments for those living with Gaucher disease for over 20 years. The company’s first approved therapy was the world’s first treatment for Gaucher disease. A few years later, Genzyme developed a next-generation enzyme replacement therapy for Gaucher disease, which is now the standard of care with unmatched years of efficacy and safety data. In 2014, Genzyme was the first to bring to market a first-line oral treatment for Gaucher disease, providing a new treatment option for patients. Genzyme will continue to serve this community and remains committed to future advancements.

About Cerdelga

Cerdelga (eliglustat), a novel glucosylceramide analog given orally, 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. The concept was initially developed by the late Norman Radin, PhD, from the University of Michigan. In pre-clinical studies, the molecule, developed with James A. Shayman, MD, also from the University of Michigan, showed specificity for glucosylceramide synthase. Following an extensive pre-clinical and early clinical research program, Cerdelga was studied in the largest Phase 3 clinical program ever conducted in Gaucher disease.

För mer information kontakta:
Tomas Gloveus, Landschef, Genzyme Sverige, 0708-19 80 73, tomas.gloveus@genzyme.com
Erik Andersson, Medicinsk chef, Genzyme Sverige, 0734 151 584, erik.andersson@genzyme.com

Om Genzyme, ett bolag i Sanofi-koncernen
Genzyme har varit en pionjär inom utveckling och framställning av banbrytande terapier för patienter med mycket ovanliga sjukdomar i över trettio år. Genzymes portfölj av behandlingar, som marknadsförs i länder över hela världen, representerar banbrytande och livräddande framsteg inom medicinen.

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