FDA Approves Cerdelga for Type 1 Gaucher Disease

Sanofi unit Genzyme says it plans to launch its new Gaucher disease therapy Cerdelga in the U.S. next month after receiving FDA approval.

The FDA cleared Cerdelga (eliglustat) as the only oral therapy for type 1 Gaucher disease patients who are missing an enzyme called glucocerebrosidase, which breaks down certain types of fat. Unlike traditional enzyme replacement therapy, which breaks down fatty deposits once they have already built up in organs such as the liver and the spleen, Cerdelga inhibits their accumulation, the company said.

The drug’s advantage over standard enzyme replacement therapy is that it’s oral, Genzyme spokeswoman Simone Azevedo said. Replacement therapy generally is done every two weeks in a hospital and takes several hours, while patients can take Cerdelga once or twice a day at home, she said.

Cerdelga was granted orphan drug designation, which applies to drugs for conditions affecting less than 200,000 people in the U.S. The agency estimates that roughly 6,000 Americans have type 1 Gaucher disease.

The approval follows two Phase III studies of nearly 200 patients who were either new to treatment or switching from enzyme replacement to Cerdelga. Results showed that Cerdelga demonstrated similar hemoglobin level, platelet count and spleen and liver volume as enzyme replacement, and better liver volume, blood platelet count and hemoglobin levels when compared with placebo, the FDA said.

The FDA and the European Medicines Agency launched a program in June to stimulate clinical trials of therapies for pediatric Gaucher disease patients. Genzyme currently has a commitment for a pediatric study in Europe that may extend to the U.S. by 2016, Azevedo said.  — Lena Freund

Originally appeared in Drug Industry Daily, the pharmaceutical industry’s number one source for regulatory news and information. Click here for more information.