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FDA Approves New Drug for Rare Genetic Disease

FDAWASHINGTON (AP) — The Food and Drug Administration on Tuesday approved a new capsule-based drug to treat Gaucher’s disease.

Regulators approved the new drug, Cerdelga, for patients with the Type 1 form of the rare genetic disorder, which causes excess fat to build up in the spleen, liver and bone marrow.

Cerdelga is from Genzyme, the specialty drugmaker that introduced the first drug for Gaucher’s disease 20 years ago. The oral treatment could serve as an alternative to Genzyme’s best-selling drug Cerezyme, which is given intravenously.

Cerezyme had sales of $935 million last year. The drug costs $300,000 for a year’s supply, on average. Genzyme says it will soon release pricing for its new drug.

Type 1 is the most common form of Gaucher’s disease and affects an estimated 6,000 people in the U.S.

Patients with Gaucher’s disease lack a certain enzyme that helps the body break down fat. Cerdelga works by reducing levels of the fat that collect in tissue and organs. It differs from Cerezyme, which is a man-made replacement for the enzyme that patients need to break down fat in the body. Cerezyme is administered by drug infusion every two weeks. The drug was introduced in 1994 and launched Genzyme into the market for rare disease drugs.

Genzyme, which is based in Cambridge, Massachusetts, is a subsidiary of French drugmaker Sanofi, which acquired the company in 2011.

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