givinostat (Duvyzat)

following a full submission assessed under the orphan medicine process:

givinostat (Duvyzat®) is accepted for restricted use within NHSScotland.

Indication under review: treatment of Duchenne muscular dystrophy (DMD) in patients 6 years of age and older.

SMC restriction: Patients who are ambulant when they initiate givinostat treatment; this includes patients who are ambulant when they initiate givinostat and become non-ambulant during treatment.

In a randomised, double-blind, phase III study, treatment with givinostat resulted in a statistically significant smaller decline in the four stairs climb time from baseline to month 18, compared with placebo.

This advice applies only in the context of an approved NHSScotland Patient Access Scheme (PAS) arrangement delivering the cost-effectiveness results upon which the decision was based, or a PAS/ list price that is equivalent or lower.

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