Kayque, Cameron, and Brian

About ELAPRASE (idursulfase)

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

How has ELAPRASE Helped Patients with MPS II?

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 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 wide range of symptoms, including difficulty breathing and difficulty walking. These symptoms may become more severe over time.

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.


➜ Learn about the safety profile of ELAPRASE

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