Elaprase

About ELAPRASE (idursulfase)

ELAPRASE is targeted to replace iduronate-2-sulfatase (I2S), the enzyme that is deficient or absent in people with Hunter syndrome.

ELAPRASE is indicated for patients with Hunter syndrome. ELAPRASE has been shown to improve walking capacity in patients 5 years and older.

In patients 16 months to 5 years of age, no data are available to demonstrate improvement in disease-related symptoms or long term clinical outcome; however, treatment with ELAPRASE has reduced spleen volume similarly to that of adults and children 5 years of age and older.

The safety and efficacy of ELAPRASE have not been established in pediatric patients less than 16 months of age.

The active substance in ELAPRASE is a formulation of idursulfase, produced by recombinant DNA technology in a human cell line. ELAPRASE is designed to replace the enzyme that is missing or defective in patients with Hunter syndrome. Without this enzyme, mucopolysaccharides – also known as glycosaminoglycans or GAGs – build up in cells, leading to tissue destruction, organ dysfunction, and other effects, and causing the signs and symptoms of Hunter syndrome.

The safety and efficacy of ELAPRASE were evaluated in a clinical study of 96 patients with Hunter syndrome. Patients in the ELAPRASE weekly treatment group exhibited a significant improvement, as compared to patients who received placebo, in the primary efficacy endpoint: a two-component score based on a statistical analysis of (1) the distance walked during a 6-minute walking test and (2) an assessment of lung function called % predicted forced vital capacity (% FVC).

When the individual components were examined separately, in an adjusted analysis, patients exhibited a 35-meter greater mean increase in the distance walked in 6 minutes compared to placebo, while the changes in % predicted FVC were not statistically significant.


> Learn about enzyme replacement therapy with ELAPRASE