voxelotor (Oxbryta)

following a full submission assessed under the orphan medicine process:

voxelotor (Oxbryta®) is accepted for restricted use within NHSScotland.

Indication under review: treatment of haemolytic anaemia due to sickle cell disease (SCD) in adults and paediatric patients 12 years of age and older as monotherapy or in combination with hydroxycarbamide.

SMC restriction: as a second line treatment for haemolytic anaemia in patients with SCD who are intolerant, ineligible or have an inadequate response to, hydroxycarbamide.

In a double-blind phase III study, voxelotor compared with placebo, increased the proportion of patients achieving an improvement in haemoglobin (Hb) levels.

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.