marstacimab (Hympavzi)

following a full submission:

marstacimab (Hympavzi®) is accepted for restricted use within NHSScotland.

Indication under review: for routine prophylaxis of bleeding episodes in patients 12 years of age and older, weighing at least 35 kg, with:

• severe haemophilia A (congenital factor VIII deficiency, FVIII < 1%) without factor VIII inhibitors, or
• severe haemophilia B (congenital factor IX deficiency, FIX < 1%) without factor IX inhibitors.

SMC restriction: severe haemophilia B (congenital factor IX deficiency, FIX < 1%) without factor IX inhibitors.

In a one-way, cross-over, open-label phase III study, marstacimab demonstrated superiority in the annualised bleeding rate of treated bleeds compared with routine FVIII or FIX prophylaxis in patients aged at least 12 years with haemophilia A or B who did not have inhibitors.

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.