FDA Approves Sanofi's Cerdelga for Gaucher Disease Type 1

The U.S Food and Drug Administration announced Tuesday that it has approved Sanofi's Cerdelga (eliglustat) to treat Type 1 form of Gaucher disease.

Gaucher disease is a rare, genetic metabolic disease in which the patient has inadequate levels of glucocerebrosidase - an enzyme that results in the buildup of fatty materials in the spleen, liver and bone marrow. Fragile bones, enlarged liver and spleen, low blood platelet count are few of the major symptoms of the disease.

The new drug is for use in adults with Gaucher's disease. Cerdelga (eliglustat) is manufactured by Sanofi's company Genzyme, which is based in Massachusetts.

Paris-based Sanofi plans to price the drug at $300,000 a year, Bloomberg reported.

Cerdelga (eliglustat) is an alternative to Sanofi's 20-year-old intravenous infusion Cerezyme, Bloomberg reported. Cerdelga works by reducing the waste produced by cell that the misisng enzyme is supposed to break down, while Cerezyme works by providing patients with an enzyme to break down the waste product.

"Today's approval offers another important treatment option for patients with Type 1 Gaucher disease," said Amy G. Egan, M.D., M.P.H., deputy director of the Office of Drug Evaluation III in FDA's Center for Drug Evaluation and Research, according to a news release. "In addition, Cerdelga received orphan drug designation from the FDA, reflecting the agency's focus and commitment to the development of treatments for rare diseases."

The safety and effectiveness of the drug were evaluated using data from 199 participants with Type 1 Gaucher disease. The team conducted two clinical trials to see whether or not the test drug was better than current treatment for the disease.

The team found that Cerdelga was effective in reducing symptoms of the disease and that it was safe to use in humans. Common side-effects of using the drug were headache, nausea, diarrhea, back pain, pain in extremities, and upper abdominal pain.

Sales of Cerezyme led to $914 million in revenue in 2013, according to data from Bloomberg.