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- A group that conducts studies on the clinical effectiveness and value of drugs found that Novartis’ (NYSE:NVS) treatment of paroxysmal nocturnal hemoglobinuria Fabhalta (iptacopan) has no net health benefit compared to the current standard of care, C5 inhibitors.
- The Clinical Institute and the Economic Review also found that Fabhalta “represents ‘low’ value for money in the long term.”
- Fabhalta, which was just approved in December, has a wholesale acquisition cost of $550,000.
- C5 inhibitors approved for PNH include Solirtis (eculizumab) and Ultomiris (ravulizumab), AstraZeneca’s (AZN) subsidiary Alexion.
- ICER found that evidence supports a net health benefit of Alexion’s danicopan, which is not yet approved in the United States, when added to a C5 inhibitor in treatment-experienced patients with clinically significant extravascular hemolysis.