FDA approves Genzyme's Cerdelga

SILVER SPRING, Md. — The Food and Drug Administration has approved Cerdelga (eliglustat) for the treatment of adult patients with the Type 1 form of Gaucher disease

The disorder — which affects about 6,000 people in the United States, according to the agency — occurs in people who don't produce enough glucocerebrosidase enzyme. This deficiency causes fatty materials to collect in the spleen, liver and bone marrow. Major symptoms of the disease include liver and spleen enlargement, anemia, low blood platelet counts and bone problems. Cerdelga works by slowing down the development of fatty materials by inhibiting the metabolic activity that forms them.

“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. “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.”

Cerdelga is manufactured by Genzyme.
 

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