NAPSR News: FDA Approves Genzyme’s Cerdelga™ (eliglustat) Capsules to Treat Gaucher

Cerdelga™ is the only first-line oral therapy approved to treat adults with Type 1 Gaucher Disease, a rare genetic disorder that affects specific cells and organs in the body.
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WASHINGTON - Aug. 20, 2014 - PRLog -- The U.S. Food and Drug Administration (FDA) has approved Cerdelga™ capsules developed by Genzyme, a Sanofi company. Cerdelga is the only first-line oral therapy established to treat certain adult Gaucher disease type 1 patients. According to the company, the therapy is expected to be available to patients within a month

“Cerdelga is an important new option for people living with Gaucher disease type 1,” said Rhonda Buyers, CEO of the National Gaucher Foundation. “As enzyme replacement therapy is the standard of treatment for Gaucher disease, patients receive regular intravenous infusions for life. With FDA's approval of a first-line oral treatment, Cerdelga has the potential to be a valuable treatment option for people living with this serious disease.”

“The approval of Cerdelga is encouraging for Gaucher patients because it is a first-line oral treatment that has demonstrated a positive risk/benefit profile, making it important from both a scientific and a clinical perspective,” said Dr. Pramod Mistry, MD, PhD, Professor of Medicine and Pediatrics and the Director of National Gaucher Disease Treatment Center at Yale University School of Medicine. “While enzyme replacement therapies break down fatty deposits that build up in cells and cause a variety of symptoms, Cerdelga inhibits the accumulation of these fatty deposits in the first place.”

FDA approval stems from clinical data achieved from two Phase 3 studies evaluating the drug’s safety and efficacy. Trial 1 was conducted involving 40 participants who had not previously received enzyme replacement therapy. Patients showed significant progress after 9 months. Reduction in spleen volume was observed, liver volume, blood platelet count, and red blood cell (hemoglobin) level also showed significant improvement, compared to the placebo.

Trial 2 involved 159 patients previously treated and stabilized on enzyme replacement therapy. The results obtained were of clinical significance as spleen volume, hemoglobin levels, platelet counts, and liver volume were stabilized.

Gaucher is a rare inherited disease affecting fewer than 10,000 people worldwide. In patients affected, they do not have enough of an enzyme, β-glucosidase (glucocerebrosidase) to break down a certain type of fat molecule called glucocerebroside. As a result the fat molecule builds up in the cells, causing lipid engorged cells and large accumulation in organs and tissues, particularly the spleen, liver and bone marrow.

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