Danicopan in paroxysmal nocturnal haemoglobinuria: a profile of its use
Declarations
Funding The preparation of this review was not supported by any external funding.
Authorship and conflict of interest N. L. France and H. A. Blair 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.
Ethics approval, Consent to participate, Consent for publication, Availability of data and material, Code availability Not applicable.
Additional information about this Adis Drug Review can be found here.
Abstract
Danicopan (Voydeya®) is a first-in-class complement factor D inhibitor which expands the treatment options available for patients with paroxysmal nocturnal haemoglobinuria (PNH) and clinically significant extravascular haemolysis (EVH). It is the third proximal complement inhibitor approved for use in PNH in Japan, the USA, EU and other countries, following the complement component 3 inhibitor pegcetacoplan and the complement factor B inhibitor iptacopan. Danicopan tablets are taken orally three times a day as an add-on to intravenous therapy with the complement component 5 inhibitors ravulizumab and eculizumab in adults. Results from ALPHA, a randomized, double-blind, placebo-controlled, international, phase 3 trial, show that danicopan significantly increases haemoglobin levels and reduces red blood cell transfusion requirements at 12 weeks. Danicopan is generally well tolerated, with adverse events typically being mild to moderate in severity. During the long-term extension of ALPHA, the rate of breakthrough haemolysis was low, and efficacy and tolerability were maintained up to 72 weeks.
© Springer Nature Switzerland AG 2025
