Learn about Elaprase

About ELAPRASE (idursulfase)

ELAPRASE is indicated for patients with Hunter syndrome (Mucopolysaccharidosis II, MPS II). 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.

Pivotal Trial Outcomes

In the pivotal trial, ELAPRASE® (idursulfase) improved walking capacity, reduced urinary GAG levels, and reduced liver and spleen volume in patients ≥5 years old.


Pivotal trial background

  • Safety and efficacy evaluated in a 53-week, randomized, double-blind, placebo-controlled clinical trial of 96 patients with Hunter syndrome, ages 5 to 31 years old
  • Patients received either:
    • ELAPRASE 0.5 mg/kg once per week (n=32)

    • ELAPRASE 0.5 mg/kg once every other week (n=32)
    • Placebo (n=32)
  • Primary efficacy outcome assessment was a 2-component composite score based on the sum of the ranks of the change from baseline to Week 53 in distance walked in 6 minutes (6-minute walk test [6-MWT]) and the ranks 
of the change in %-predicted forced vital capacity (FVC)
  • Pharmacodynamic assessments included urinary GAG levels and changes in liver and spleen volume

Achieved Primary Endpoint

The composite primary endpoint differed statistically significantly between the 3 groups, and the difference was greatest between the placebo group and the once-weekly treatment group (once-weekly ELAPRASE vs placebo, P=0.0049)

Improved Walking Capacity

  • The weekly ELAPRASE-treated group experienced a greater increase in the distance walked in 6 minutes compared to the placebo-treated group

Pulmonary Function

  • The changes from baseline to Week 53 in %-predicted FVC were not statistically significant

Improved Walking Capacity

Elaprase pulmonary function endpoint

  • The weekly ELAPRASE-treated group experienced a greater increase in the distance walked in 6 minutes compared to the placebo-treated group

Pulmonary Function

  • The changes from baseline to Week 53 in %-predicted FVC were not statistically significant

Reduced Urinary GAG Levels

Elaprase Reduced Urinary GAG levels

  • Mean urinary GAG levels were reduced in the ELAPRASE once-weekly group
  • Urinary GAG levels still remained above the upper limit of normal in half of the ELAPRASE-treated patients
  • Urinary GAG levels remained elevated and essentially unchanged in the placebo group
  • The responsiveness of urinary GAG levels to dosage alterations of ELAPRASE is unknown, and the relationship of urinary GAG to other measures of clinical response has not been established
  • Patients who tested positive for antiidursulfase antibodies (Ab) experienced a less-pronounced decrease in urinary GAG levels
References: 1. Muenzer J, et al. Genet Med. 2006;8(8):465-473.

Reduced Liver and Spleen Volumes

Elaprased reduced liver and spleen volumes

  • Sustained reductions in both liver and spleen volumes were observed in the ELAPRASE once-weekly group through Week 53 compared to the placebo group
  • There were essentially no changes in liver and spleen volumes in the placebo group
References: 1. Muenzer J, et al. Genet Med. 2006;8(8):465-473.

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How Does
ELAPRASE Work?

ELAPRASE is designed to replace I2S, the enzyme that is deficient or absent in people with Hunter syndrome.

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Questions About
ELAPRASE?

Contact OnePath® for personalized support, insurance help, co-pay assistance, education and more.

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Important Forms
& Resources

Access our directory of helpful ELAPRASE resources for patients and families.