TUESDAY, July 25 (HealthDay News) -- The U.S. Food and Drug Administration has approved Elaprase (idursulfase), the first product for the treatment of Hunter syndrome. Designated as an orphan product by FDA, Elaprase is a new molecular entity that contains an active ingredient never before marketed in the United States.
FDA approval was based on a randomized, double-blind, placebo-controlled study of 96 patients with Hunter syndrome in which treated participants had an improved capacity to walk. Upon completion of a 53-week trial, those who received Elaprase infusions walked an average 38-yard greater distance in six minutes compared with patients who received placebo.
Serious adverse events included potentially life-threatening hypersensitivity reactions. Appropriate medical support should be readily available when Elaprase is administered because of the potential for severe hypersensitivity reactions. Patients and their physicians are encouraged to participate in a voluntary Hunter Outcome Survey, which will monitor and evaluate the safety and effects of long-term Elaprase treatment.
Elaprase is manufactured by Shire Human Genetic Therapies, Inc., in Cambridge, Mass. Wire reports suggest that Shire expects to market the drug for $300,000 a year.
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