Elaprase (idursulfase) is the first and only treatment approved for people suffering from Hunter syndrome, a rare, life-threatening genetic condition also known as mucopolysaccharidosis II. The product, which is given as weekly infusions, replaces the missing enzyme that Hunter syndrome patients fail to produce in sufficient quantities.
In a 53-week phase II/III trial, Elaprase demonstrated that it can provide clinically important benefits to Hunter syndrome patients. In this trial, the primary efficacy endpoint of the trial was a composite analysis of changes from baseline in two clinical measures: a six-minute walk test and percent predicted forced vital capacity. After one year of treatment, patients receiving weekly infusions of Elaprase experienced a mean increase in the distance walked in six minutes of 35 meters compared to patients receiving placebo.
Shire expects to launch Elaprase in the US within the next 30 days. The company has also submitted a marketing authorization application (MAA) for Elaprase to the European Medicines Agency (EMEA). Based on average evaluation times, Shire anticipates completion of the EMEA review by year end.
“Regulatory approval of Elaprase will enable physicians to move needy patients beyond palliative care and make Hunter syndrome a treatable disease,” said Dr Joseph Muenzer of the University of North Carolina at Chapel Hill. “Until today, there were no options for addressing the underlying cause of this devastating disease.”