Shire plc announced that the US FDA has granted 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.
Elaprase is the first and only treatment approved for people suffering from Hunter syndrome. The product, which is given as weekly infusions, replaces the missing enzyme that Hunter syndrome patients fail to produce in sufficient quantities.
Shire expects to launch Elaprase in the United States within the next 30 days.
"The FDA approval of Elaprase marks a significant milestone in Shire's effort to provide meaningful treatments for patients suffering from genetic diseases," said Matthew Emmens, chief executive officer of Shire. "A hallmark of the Elaprase program is the commitment demonstrated by patients and families, investigators and Shire employees involved in the development effort. We look forward to making Elaprase available to patients in the coming weeks."
"Regulatory approval of Elaprase will enable physicians to move needy patients beyond palliative care and make Hunter syndrome a treatable disease," said Joseph Muenzer, MD., Ph.D, of the University of North Carolina at Chapel Hill. "Until today, there were no options for addressing the underlying cause of this devastating disease."
Shire submitted a Marketing Authorization Application (MAA) for Elaprase to the European Medicines Agency (EMEA) on December 1, 2005. Based on average evaluation times, Shire anticipates completion of the EMEA review by year end. In European countries that have mechanisms for pre-approval access, Shire has also submitted applications.
Elaprase is a purified form of the lysosomal enzyme iduronate-2-sulfatase and is produced by recombinant DNA technology in a human cell line.
In conjunction with the market approval of Elaprase, Shire Human Genetic Therapies (the Shire business unit focused on genetic diseases) has introduced a new product support centre called OnePathSM for the US market. OnePathSM is a single source of product support for healthcare providers, patients and their families, where personalized, comprehensive information about Elaprase is available from a case manager. Case managers can provide information about coding, reimbursement and insurance verification, authorization letters, product access and treatment centre locations. OnePathSM also offers education about Hunter syndrome and can refer patients to additional support services, if needed.
Shire Human Genetic Therapies is actively tracking health data among individuals affected by Hunter syndrome as part of the company's long-term outcome survey, called the Hunter Outcome Survey (HOS). HOS is designed to support the gathering, analysis, reporting and sharing of data from around the world about Hunter syndrome. Shire believes that the inclusion of all people affected by Hunter syndrome and the analysis and dissemination of this information will allow for further understanding of Hunter syndrome and disease education on a global scale.