Iptacopan in paroxysmal nocturnal haemoglobinuria: a profile of its use

Funding The preparation of this review was not supported by any external funding.

Authorship and conflict of interest M. B. Brown and M. Shirley are salaried employees of Adis International Ltd/Springer Nature and declare no relevant conflicts of interest. All authors contributed to this article and are responsible for its content.

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Additional information about this Adis Drug Review can be found here.

Abstract

Iptacopan (Fabhalta®), a complement factor B inhibitor, is a valuable treatment option for adult patients with paroxysmal nocturnal haemoglobinuria (PNH). It is the first oral monotherapy available for treatment of PNH in the USA and the EU. As demonstrated in the phase III clinical trials APPLY-PNH and APPOINT-PNH, twice daily iptacopan provided clinically meaningful improvements in haemoglobin levels in both anti-complement component 5 (anti-C5) therapy-experienced and complement inhibitor-naïve patients with PNH and anaemia. In APPLY-PNH, iptacopan was superior to continuing anti-C5 therapy at improving haemoglobin levels without the need for red blood cell transfusions (RBCTs). In both trials, iptacopan also reduced the need for RBCTs and reduced patient-reported fatigue levels. Iptacopan was generally well tolerated, with the most common adverse events being acute mild-to-moderate headache and with no discontinuations due to adverse events recorded in either trial. Currently available data show iptacopan efficacy is durable over 48 weeks; longer term efficacy and safety data are awaited with interest.

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