ICER finds benefit evidence for Novartis and Alexion PNH drugs not adequate

14 March 2024
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US health technology assessor the Institute for Clinical and Economic Review (ICER) released a Final Evidence Report assessing the comparative clinical effectiveness and value of Novartis’ (NOVN: VX) iptacopan and AstraZeneca (LSE: AZN) subsidiary Alexion’s danicopan for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).

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.

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