ELAPRASE is designed to replace I2S, the enzyme that is deﬁcient or absent in people with Hunter syndrome.
Patients with Hunter syndrome do not produce a sufficient amount of an enzyme called iduronate-2-sulfatase. This enzyme is needed to break down substances in the body called glycosaminoglycans (GAGs). Since patients with Hunter syndrome cannot break these substances down, the GAGs gradually build up in most of the organs in the body and can damage them. This causes a range of disease-related signs and symptoms, including diminished lung function and decreased walking capacity.
In a clinical study of 96 people age 5 or older with Hunter syndrome, ELAPRASE was shown to significantly increase patients’ ability to walk farther compared to those who received an infusion of placebo.
ELAPRASE was also shown to improve some other measures of activity such as GAG levels in the urine, and the size of the liver and spleen. The results of tests on a measure of lung capacity, known as the % predicted forced vital capacity, or % FVC, were not significant.