Shire submits Biologics License Application to FDA for Elaprase

By Ted A. Knutson

Washington, Nov. 28 - Shire plc, the holding company of Shire Pharmaceuticals Group plc, has submitted a Biologics License Application to the U.S. Food and Drug Administration for idursulfase under the trade name Elaprase, formerly referred to as I2S, the company said in a news release Friday.

If approved, Elaprase would be the first human enzyme replacement therapy for the treatment of Hunter syndrome, also known as Mucopolysaccharidosis II (MPS II).

Idursulfase has previously received fast track designation from the FDA, and Shire has requested priority review of this submission, which would result in a six-month review.

In addition, submission to the European Medicines Agency is anticipated before the end of 2005 and typically takes 12 months for review and approval.

Shire said the license application contains results of the pivotal AIM (Assessment of I2S in MPS II) study, which studied 96 patients over 52 weeks, and is the largest study conducted for a Mucopolysaccharidosis disorder.

According to the study, patients who received 0.5mg/kg of Elaprase on a weekly basis showed a statistically significant difference in the primary efficacy endpoint compared with patients receiving a placebo. Additional data demonstrated improvements in key secondary endpoints.

Treatment with Elaprase was generally well tolerated by patients in the trial. The most common adverse events observed were associated with the clinical manifestations of Hunter syndrome. Of the adverse events considered possibly related to Elaprase, infusion-related reactions were the most common and were generally mild, the company said.

There were two patient deaths during the study, both of which were considered to be unrelated to treatment with Elaprase. No patient withdrew from the trial due to an adverse event considered related to Elaprase, and to date all patients have agreed to continue in the extension study.

Shire plc, a Basingstoke, England-based pharmaceutical company, is focused on drug development for the central nervous system.

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