FDA approves new drug for rare genetic disease
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.
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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