Sanofi's Cerdelga Shows Promise in Late Stage Trial

Published: Feb 18, 2015

 Actavis (ACT) Touts Potential $6 Billion Pipeline at Investor Meeting
February 18, 2015
By Jessica Wilson, BioSpace.com Breaking News Staff

Genzyme Corporation said today that its Phase III trial of Gaucher drug Cerdelga had met all primary endpoints in treating the rare genetic disorder, and reduced spleen size by as much as 28 percent.

Genzyme, a Sanofi company, published the results of the Phase III study, called ENGAGE, evaluating its Type-1 Gaucher drug Cerdelga (eliglustat) in treatment-naïve patients in yesterday’s issue of The Journal of the American Medical Association.

It found the drug met its endpoints of measured change in the symptoms associated with Gaucher, a disease that causes lipid accumulation in the bone marrow, lungs, spleen, liver and the brain, which leads to spleen and liver enlargement, red and white blood cell abnormalities and bone deterioration.

The study involved 40 patients divided into groups based on spleen volume. Patients were then randomized to receive Cerdelga or a placebo for nine months. Patients who received Cerdelga experienced a statistically significant spleen size reduction of 28 percent, while the placebo group only saw a reduction of two percent.

Cerdelga also achieved statistically significant results as measured by secondary endpoints. Cerdelga increased hemoglobin levels, decreased liver volume and increased platelet levels. In addition, patients who received Cerdelga did not experience any serious adverse reactions.

“We are very encouraged by these results as they ultimately point to a safe and effective oral treatment option for patients living with Gaucher disease,” said lead author Pramod Mistry, professor of pediatrics and internal medicine at Yale University School of Medicine, in a statement.

Mistry also spoke to Healio, a medical blog targeted at clinical health practitioners, about the study.

“The study is important because it represents the first ever randomized placebo-controlled clinical trial of a treatment for type 1 Gaucher disease,” Mistry told Healio.

The U.S. Food and Drug Administration (FDA) approved Cerdelga in August 2014, largely based on these study results. The European Commission (EC) granted marketing authorization for Cerdelga in January 2015.

In the wake of the EC’s decision, analyst Trevor Lowenthal at Seeking Alpha weighed in on the significance of Cerdelga for Genzyme and Sanofi. The EC decision is a bell weather of the way future approval decisions will fall throughout the world—indicating that Cerdelga will be approved in large markets such as Canada, India and China, according to Lowenthal.

Based on the likelihood of future approvals, the $310,000 price tag of Cerdelga and the fact that it’s the only oral treatment for Gaucher, Lowenthal concluded that, “Sanofi's Gaucher treatments should have the potential to generate over $1 billion in revenues.”



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