Interventions of Interest

  • iptacopan (Fabhalta ®, Novartis)
  • danicopan (Voydeya, Alexion Pharmaceuticals)

The independent appraisal committee voted that the current evidence is not adequate to demonstrate a net health benefit for iptacopan over a C5 inhibitor, although use in treatment-experienced patients with clinically significant extravascular hemolysis received more favorable votes. The committee also found that at current pricing, iptacopan represents “low” long-term value for money.

Furthermore, the committee voted that the evidence is adequate to demonstrate a net health benefit for add-on danicopan compared to continuing C5 inhibitor alone in treatment-experienced patients on a stable C5 Inhibitor regimen with clinically significant extravascular hemolysis. Danicopan’s price is not yet known; however, ICER analyses suggest danicopan would achieve common thresholds for cost-effectiveness if priced between $12,300 to $13,100.   

Final Documents

ICER’s Vice President of Research, Foluso Agboola, MBBS, MPH:

“PNH is a rare, acquired blood disorder that primarily manifests in fatigue, and if severe, requires lifelong dependence on blood transfusions. “Iptacopan (as an alternative to C5 inhibitor therapy) and danicopan (as an add-on to a C5 inhibitor) are promising new oral options for PNH patients. As indicated by the votes from the independent appraisal committee, the current evidence was judged more favorably for add-on danicopan in treatment-experienced PNH patients with anemia; however, there are still uncertainties around the long-term benefits of both therapies. The discussion during the public meeting highlighted the impact of high costs associated with C5 inhibitors – the current standard of care for PNH – on accessibility and affordability, and its effect on the pricing of new PNH therapies.”

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