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news.Shire Pharmaceuticals has submitted a biologics license application with the FDA for Elaprase, a human enzyme replacement therapy for the treatment of Hunter syndrome - a rare, life threatening, genetic disorder with no currently available treatment.
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Elaprase has previously received fast-track designation from the FDA, and Shire has requested priority review of this submission, which should result in a decision being made by the FDA within six months. Submission to the European Medicines Agency (EMEA) is anticipated before the end of 2005 and typically takes 12 months for review and approval.
Individuals with Hunter syndrome lack the enzyme iduronate-2-sulfatase, which is essential in the continuous process of replacing and breaking down glycosaminoglycans (GAG). As a result, GAG remains stored in cells in the body causing progressive damage.
As the disease progresses, a variety of symptoms appear including enlarged liver and spleen, heart failure, decreased endurance, obstructive and restrictive airway disease, sleep apnea, joint stiffness, and, in some cases, central nervous system involvement.
If central nervous system involvement exists, the life expectancy for patients with Hunter syndrome is typically 10-15 years of age, however, some patients can survive into the fifth or sixth decade of life.
“We are now a significant step closer to helping patients and their families living with Hunter syndrome, and I am pleased that this application has been submitted on schedule and I look forward to approval and subsequent launch in 2006,” said Dr David Pendergast, executive vice president and general manager of Shire human genetic therapies.