UK pharmaceutical group Shire is one step closer to getting its Elaprase (idursulfase; formerly known as I2S) approved in the USA as the first human enzyme replacement therapy for the treatment of Hunter syndrome, after filing a Biologics License Application with the US Food and Drug Administration.
Hunter syndrome, also known as MPS II, is a rare, life-threatening genetic disorder with no available treatment. As the disease progresses, a variety of symptoms appear including, enlarged liver and spleen, and heart failure. Consequently, Shire has requested that this submission will be given a priority review, which would result in a decision within six months of filing.
Commenting on the development, David D Pendergast, executive vice president of Shire Human Genetic Therapies, said: “Our filing with the FDA is a milestone for Shire and our team, who have been steadfast in the research and development of this much needed treatment. 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.”
The company expects a similar submission to the European Medicines Agency before the end of year, with a regulatory decision within 12 months of filing.