FDA Approves Genzyme Corporation's Gaucher Disease Pill Cerdelga

Published: Aug 20, 2014

FDA Approves Genzyme Corporation's Gaucher Disease Pill Cerdelga FDA Approves Genzyme Corporation's Gaucher Disease Pill Cerdelga

August 20, 2014
By Krystle Vermes, BioSpace.com Breaking News Staff

Cambridge-based Genzyme Corporation , a biotechnology company, announced on Aug. 19 that the U.S. Food and Drug Administration has approved of Cerdelga, its drug for Gaucher disease type 1 patients.

The drug, which comes in a capsule, is now the only form of oral therapy for the condition. Some patients who metabolize Cerdelga at a rapid or undetermined rate may not be eligible for the drug. However, Cerdelga is set to become available to other patients within one 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 FDA approved the drug after receiving efficacy data from two positive Phase 3 studies. By the end of the studies, the adverse reactions were limited to fatigue, headache, nausea, diarrhea, back pain, pain in extremities, and upper abdominal pain in patients who received Cerdelga.

Cerdelga reduces the production of glucosylceramide, the substance that builds up in the cells and tissues of people with Gaucher disease.

“More than twenty years ago, Genzyme introduced the world’s first treatment for Gaucher disease,” said said Genzyme President and CEO, David Meeker. “We are proud to build on this legacy and continue to improve Gaucher patients’ lives through ongoing research and new therapies. The approval of Cerdelga represents our unwavering commitment to the Gaucher patient community.”

Gaucher disease impacts approximately 10,000 people worldwide. Those who have the condition are lacking glucocerebrosidase, an enzyme that is designed to break down fat molecules within the body. In turn, lipid engorged cells build up in areas of the body such as the spleen and liver. This can result in excessive bleeding, bruising and bone disease in patients.

“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 Pramod Mistry, M.D., Ph.D., 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.”

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