iptacopan (Fabhalta)

following a full submission:

iptacopan (Fabhalta®) is accepted for restricted use within NHSScotland.

Indication under review: As monotherapy in the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have haemolytic anaemia.

SMC restriction: under the advice of the national PNH service.

In an open-label phase III study in patients with PNH who had persistent anaemia despite treatment with anti-C5 treatment iptacopan significantly increased the proportion of patients whose haemoglobin levels improved by at least 2 g/dL and the proportion of patients with haemoglobin levels greater than or equal to 12 g/dL, compared with anti-C5 treatment. In a single-arm phase III study in patients who had not received anti-C5 treatment, 92% of patients had an increase in haemoglobin of at least 2 g/dL after 24 weeks.

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.