U.S. FDA approves Sanofi's Gaucher disease drug Cerdelga

by Reuters
Tuesday, 19 August 2014 21:29 GMT

Aug 19 (Reuters) - The U.S. Food and Drug Administration said on Tuesday it has approved Sanofi SA's drug Cerdelga to treat patients with type 1 Gaucher disease, a rare genetic disorder.

The drug was developed by Sanofi's Genzyme unit, which also developed the Gaucher disease drug Cerezyme. Cerdelga, known also as eliglustat, is a pill, while Cerezyme must be infused.

Gaucher disease is caused by deficient levels of an enzyme needed to break down certain fats, leading to enlarged liver and spleen. Symptoms include bone pain, osteoporosis, anemia and fatigue.

Dr. David Meeker, Genzyme's chief executive, said in an interview that the company plans to price the drug on a par with Cerezyme, which has an average annual cost of about $300,000.

Because Cerezyme is dosed in units per kilograms of body weight, the cost per patient varies. The pill will typically be taken twice a day, regardless of body weight.

"The challenge has been to think about pricing in a way that does not drive medical decision-making," Meeker said. "Our goal here is not to drive patients one way or another."

The two drugs work differently. Cerezyme is an enzyme replacement therapy and accounts for about 70 percent of the Gaucher market. The new drug works by reducing the amount of fatty lipids produced by the body. The two drugs are similarly effective, Meeker said.

Meeker said he cannot estimate how many patients will switch, noting that there are some 7,000 patients being treated worldwide and there may be 10,000 patients in total.

There are also drugs to treat Gaucher made by other companies. Pfizer Inc and Protalix Biotherapeutics Inc make the drug Elelyso, while Shire Plc makes the drug Vpriv.

Type 1 Gaucher disease is particularly prevalent among Ashkenazi Jews. (Reporting by Toni Clarke in Washington; Editing by Leslie Adler)

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