ELAPRASE (idursulfase), enzyme replacement

RARE DISEASE - Focus

Opinions on drugs - Posted on Jul 30 2015

Re-assessment of the Actual Benefit and of the improvement in actual benefit

ELAPRASE has Marketing Authorisation in the long-term treatment of patients with Hunter syndrome (mucopolysaccharidosis type II).
It is the only enzyme replacement therapy available for managing these patients.
Its short-term efficacy has been demonstrated on the 6-minute walk test (6MWT) versus placebo (52 weeks). There are no data showing neurological improvement on treatment.
There are insufficient data to establish its medium and long-term efficacy. Given the long-term results in real life, one may question the relevance of the criteria used to assess the long-term efficacy of idursulfase treatment.

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Therapeutic use

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