
Md Babul Hosen/iStock via Getty Images
- A group that conducts clinical effectiveness and value studies on drugs found that Novartis’ (NYSE:NVS) paroxysmal nocturnal hemoglobinuria treatment Fabhalta (iptacopan) does not have a net health benefit over the current standard of care, C5 inhibitors.
- The Institute for Clinical and Economic Review also found that Fabhalta “represents ‘low’ long-term value for money.”
- Fabhalta, which was just approved in December, comes with a wholesale acquisition cost of $550,000.
- C5 inhibitors approved for PNH include AstraZeneca (AZN) subsidiary Alexion’s Solirtis (eculizumab) and Ultomiris (ravulizumab).
- ICER did find that evidence supported a net health benefit for Alexion’s danicopan, which is not yet approved in the U.S., when added on to a C5 inhibitor in treatment-experienced patients with clinically significant extravascular hemolysis.

