elosulfase alfa (Vimizim)

following a full submission considered under the ultra-orphan medicine process

elosulfase alfa (Vimizim®) is not recommended for use within NHS Scotland.

Indication under review: treatment of mucopolysaccharidosis, type IVA (Morquio A Syndrome, MPS IVA) in patients of all ages.

In a double-blind placebo-controlled study the difference from baseline in the mean distance walked  in the 6-minute walking test was significantly longer for elosulfase alfa, given weekly, than placebo at week 24.

The submitting company’s justification of the treatment’s cost in relation to its health benefits was not sufficient and in addition the company did not present a sufficiently robust economic analysis to gain acceptance by SMC.

This advice takes account of the views from a Patient and Clinician and Engagement (PACE) meeting.