News Feature | August 21, 2014

FDA OKs Sanofi Genzyme's Cerdelga For Gaucher Disease

By Estel Grace Masangkay

Sanofi company Genzyme announced that the U.S. Food and Drug Administration (FDA) has approved Cerdelga (eliglustat) capsules as treatment for certain adult patients with type 1 Gaucher disease.

Cerdelga is a specific ceramide analogue inhibitor of glucosylceramide synthase with broad tissue distribution. The drug is manufactured as a hard gelatin oral capsule containing eliglustat and is indicated for the long-term treatment of rare genetic disease Gaucher disease type 1 (GD1), approved under its orphan drug designation. The newly approved pill offers an alternative to Sanofi’s 20-year old intravenous drug Cerezyme for Gaucher disease.

Gaucher disease is an inherited condition that affects approximately 6,000 people in the U.S. The disease is caused by the decreased production of an enzyme known as glucocerebrosidase, the lack of which results in the buildup of fatty material in the spleen, bone marrow, and liver. Symptoms include enlargement of the liver and spleen, anemia, low blood platelet counts, and problems in the bone.

“Today’s approval offers another important treatment option for patients with Type 1 Gaucher disease,” said Amy G. Egan, deputy director of the Office of Drug Evaluation III in FDA’s Center for Drug Evaluation and Research.

The approval was supported by positive safety and efficacy results of two clinical trials investigating Cerdelga in 199 patients with Gaucher disease type 1. As the trial’s primary endpoint, the drug achieved a greater reduction in spleen volume from baseline compared to placebo. Patients who were treated with Cerdelga also achieved greater improvement in liver volume, red blood cell level, and blood platelet count.

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

According to Bloomberg, Sanofi plans to sell Cerdelga at approximately $300,000 a year, which it calculated to be the average annual cost of Cerezyme infusions once every two weeks. “Our goal is to price this in a way that does not bias the medical decision-making process,” said Meeker.