etranacogene dezaparvovec (Hemgenix)

following a full submission assessed under the orphan medicine process:

etranacogene dezaparvovec (Hemgenix^®) is accepted for use within NHSScotland on an interim basis subject to ongoing evaluation and future reassessment.

Indication under review: for the treatment of severe and moderately severe haemophilia B (congenital factor IX deficiency) in adult patients without a history of factor IX inhibitors.

In an open-label, non-randomised, single-arm, phase III study, the annualised bleeding rate was reduced following treatment with etranacogene dezaparvovec compared with a lead-in period of regular factor IX prophylaxis.

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.