Shire: Trial results positive for idursulfase to treat rare Hunter syndrome

By E. Janene Geiss

Philadelphia, Oct. 28 - Shire Pharmaceutical Group plc announced Friday that further results from a clinical trial evaluating the investigational human enzyme replacement therapy, idursulface, for the treatment of Hunter syndrome show statistical improvement in patients' symptoms.

The positive results, presented at the annual meeting of the American Society of Human Genetics, furthers the hope that idursulface could be the first treatment option for this rare, debilitating and fatal disease, officials said in a company news release.

The primary efficacy endpoint of the trial was a composite of two clinical measures - forced vital capacity and the 6-minute walk test. Patients receiving the every-other-week dosing regimen of idursulfase showed a statistically significant difference compared to a placebo. The treatment also was well tolerated, officials said.

"Data from this study are very encouraging for patients and families living with Hunter syndrome. I am excited about the potential of idursulfase as the first treatment option for patients, once approved," Joseph Muenzer of the University of North Carolina at Chapel Hill, said in the release. Muenzer presented the results at the conference.

Shire said it expects to file for regulatory approval of idursulfase in both the United States and Europe in the fourth quarter of 2005. It currently has orphan drug status in the United States and European Union, officials said.

Shires said it believes there are about 2,000 patients worldwide afflicted with Hunter syndrome in countries where reimbursement may be possible.

The company also announced Friday that results from a study of the Attention Deficit/Hyperactivity Disorder drug Adderall XR show symptom improvements in adults.

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

© 2015 Prospect News.
All content on this website is protected by copyright law in the U.S. and elsewhere. For the use of the person downloading only.
Redistribution and copying are prohibited by law without written permission in advance from Prospect News.
Redistribution or copying includes e-mailing, printing multiple copies or any other form of reproduction.