eliglustat (Cerdelga)

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

eliglustat (Cerdelga®) is accepted for use within NHS Scotland.

Indication under review: for the long-term treatment of adult patients with Gaucher disease type 1 (GD1) who are CYP2D6 poor metabolisers, intermediate metabolisers or extensive metabolisers.

In a phase III, randomised, controlled study in patients with GD1 who were previously stabilised on enzyme replacement therapy (ERT), comparable proportions of patients treated with eliglustat versus ERT maintained stability of haemoglobin concentration, platelet count, spleen and liver volumes at a non-inferiority margin of 25% in the protocol-specified analysis.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of eliglustat. This advice is contingent upon the continuing availability of the PAS in NHS Scotland or a list price that is equivalent or lower.
This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.