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Funding drugs for rare diseases
Shire plc’s drug to treat Gaucher disease has been approved by the U.S. Food and Drug Administration, the company announced today. The drug was fast-tracked by regulators, due to a shortage of the only approved drug for Gaucher disease, Genzyme Corp.’s Cerezyme.
Shire’s drug, now called VPRIV, has been prescribed to patients before its approval on an emergency basis for several months due to the shortage.
U.K.-based Shire, which has two facilities in Massachusetts, has announced that it will initially price its drug at approximately 15 percent less than Genzyme’s drug, which has an annual cost of over $200,000 per patient.
Gaucher disease affects only one in every 50,000 people. It causes enlargement of the spleen and liver, among other symptoms and can be fatal.
Cambridge-based Genzyme had to shut down its Allston manufacturing plant for six weeks last summer due to the discovery of a virus.
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