Top 5 PNH stories of 2025
Most-read stories focused on treatments, diagnosis, and patient experience
In 2025, PNH News delivered timely coverage of the latest research developments and advances in care related to paroxysmal nocturnal hemoglobinuria (PNH). To close the year, we’ve compiled the five most-read stories. As we move into 2026, we remain committed to supporting the PNH community with clear reporting and patient-centered insights.
No. 5 — Symptoms burdensome despite treatment, interviews find
In interviews, U.S. adults with PNH reported fatigue that makes it harder to get through daily tasks, stay socially engaged, and keep mentally focused, even while on treatment. The findings suggest that doctors may need to take a closer look at how PNH symptoms affect daily life, not only in newly diagnosed patients but also in those who have been on treatment for years.
Read more here.
No. 4 — Patients highly satisfied with Fabhalta
Interviews with patients in eight countries found that many were highly satisfied with the effectiveness and ease of use of Novartis’ Fabhalta (iptacopan). Patients reported having more energy and fewer breakthrough symptoms after switching to the oral therapy, which is taken twice daily and works by blocking the overactive complement system that destroys red blood cells in PNH. Many also appreciated not needing frequent infusions, finding the capsules more convenient for work, travel, and daily life. While experiences varied, most felt Fabhalta was a clear improvement over previous treatments.
Read more here.
No. 3 — AI models aim to speed up PNH diagnoses
Atropos Health is collaborating with Novartis to develop artificial intelligence (AI) models that may help identify people with rare diseases such as PNH who not yet been diagnosed. By training these tools on real-world data, the goal is to shorten the time between first symptoms and diagnosis and treatment. More accurate AI models may reduce guesswork, allowing patients to access testing sooner and helping doctors deliver faster, more informed care.
Read more here.
No. 2 — Pozelimab-cemdisiran combo better than Ultomiris in clinical trial
An experimental combination of pozelimab and cemdisiran may control intravascular hemolysis — the breakdown of red blood cells within blood vessels — more effectively than Ultomiris (ravulizumab-cwvz) in adults with PNH. A Phase 3 trial and its extension are testing the treatment in patients who are either new to complement inhibitors or have not recently used them. The combination is given as an under-the-skin injection every four weeks, and so far, has reduced hemolysis compared with Ultomiris, which is administered by intravenous infusion every eight weeks.
Read more here.
No. 1 — Fabhalta expected to save nearly $4M in lifetime costs
Fabhalta could save about $4 million in lifetime costs compared with standard PNH treatments that are given by infusion. In clinical trials supporting its approval, the oral therapy was not only more convenient for patients but also more effective at controlling PNH symptoms. Researchers from Yale School of Medicine in Connecticut and the University of Pennsylvania in Philadelphia estimated Fabhalta’s annual cost at $488,220. Over a patient’s lifetime, Fabhalta would deliver 12.6 quality-adjusted life years at a cost $9.52 million, compared with $13.5 million for standard treatments providing 10.8 quality-adjusted life years.
Read more here.
We hope our reporting at PNH News throughout 2025 has been informative and meaningful for our readers. As we move into 2026, we remain dedicated to serving everyone affected by PNH and we wish all our readers a healthy and happy new year!
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