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FDA approves new drug for rare genetic disease
Pharmaceuticals » Genzyme product attacks disease that causes fat to build in organs.
First Published Aug 19 2014 06:11 pm • Last Updated Aug 19 2014 06:11 pm

Washington • The Food and Drug Administration has 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.

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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.




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