Shire gets FDA approval to sell Elaprase for Hunter syndrome

By Elaine Rigoli

Tampa, Fla., July 24 - The Food and Drug Administration has granted Shire plc marketing approval for Elaprase, a human enzyme replacement therapy for the treatment of Hunter syndrome, also known as Mucopolysaccharidosis II (MPS II).

Hunter syndrome is a rare, life-threatening genetic condition that results from the absence or insufficient levels of the lysosomal enzyme iduronate-2-sulfatase. Without this enzyme, cellular waste products accumulate in tissues and organs, which then begin to malfunction.

Shire expects to launch Elaprase in the United States within the next 30 days.

Shire submitted a Marketing Authorization Application for Elaprase to the European Medicines Agency on Dec. 1, 2005. Based on average evaluation times, Shire said it anticipates completion of the review by the end of 2006.

A 53-week, randomized, double-blind, placebo-controlled phase 2/ 3 trial demonstrated that Elaprase provides clinically important benefits to Hunter syndrome patients, the company said in a news release.

The primary efficacy endpoint of the trial was a composite analysis of changes from baseline in two clinical measures: a six-minute walk test and percent predicted forced vital capacity.

Shire said this endpoint achieved statistical significance compared to a placebo.

After one year of treatment, patients receiving weekly infusions of Elaprase experienced a mean increase in the distance walked in six minutes of 35 meters compared to patients receiving a placebo, the company added.

Shire is a pharmaceutical company based in Basingstoke, U.K.

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