ELAPRASE is a prescription medicine for patients with Hunter syndrome. ELAPRASE has been shown to improve walking ability in patients 5 years and older.
In patients 16 months to 5 years old, ELAPRASE did not show improvement in disease-related symptoms or long term clinical result; however, treatment with ELAPRASE has reduced spleen size similarly to patients 5 years and older.
It is not known if ELAPRASE is safe and effective in children under 16 months old.
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.