FDA formally approves new drug for Gaucher disease

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The Food and Drug Administration on Friday formally approved a new drug called velaglucerase alfa for injection (tradenamed VPRIV) for treatment of the rare genetic disorder Gaucher disease. The agency has already been allowing the company to ship the drug to some patients in the United States because of a shortage of Cerezyme, the only drug now approved for the condition. Cerezyme has been available in only limited quantities because of manufacturing problems at Genzyme Corp.’s Alston, Mass. manufacturing plant.

Gaucher, which affects as many as 1 in 40,000 Americans, is caused by the lack of an enzyme called glucocerebrosidase, which breaks down a lipid called glucocerebroside. Without this enzyme, harmful amounts of glucocerebroside build up in the liver, spleen, bones, bone marrow and nervous system, preventing cells and organs from working properly. There are three types of Gaucher. Those with the mildest form have a small decrease in life expectancy. Those with the moderate form generally die before age 30, and those with the most severe form typically die before age 3. Both drugs are infused enzymes that can break down the cerebroside and prevent it from accumulating.

Cerezyme is one of the most expensive drugs on the U.S. market, costing patients an estimated $200,000 or more a year. Shire of Cambridge, Mass., which manufactures VPRIV, said Friday that it will undercut Genzyme’s price by 15% in an effort to gain market share. The company said it also has a program to help insured patients cover their co-payments.

-- Thomas H. Maugh II