EXTON, PA, March 18, 2025 (GLOBE NEWSWIRE) -- Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare and debilitating blood disorder that affects approximately 8,000 to 10,000 people in North America and Western Europe.1 Patients often suffer from severe fatigue, and left untreated, PNH can lead to serious complications, including hemolytic anemia, chronic kidney disease, and life-threatening thrombosis.2 While complement 5 (C5) inhibitors such as Soliris and Ultomiris have long been the foundation of treatment, the PNH landscape is evolving rapidly, with new therapies and emerging biosimilars reshaping physician decision-making and patient care strategies.
Spherix Global Insights recently released its latest findings from RealTime Dynamix™: Paroxysmal Nocturnal Hemoglobinuria Q1 2025 (US) an independent tracking study based on insights from 51 U.S. hematologists with PNH patients. The research reveals a rising sense of urgency among physicians to initiate treatment, with growing consensus that all PNH patients should be on a complement or factor inhibitor, regardless of disease burden. With the expansion of oral treatment options, adherence is also becoming a stronger driver in prescribing decisions.
The competitive landscape for PNH has seen significant shifts, particularly with the introduction of Fabhalta (Novartis), which launched in December 2023, alongside newer market entrants Voydeya (Alexion/AZ) and PiaSky (Genentech/Roche), offering new mechanisms for treatment. In just over a year, Fabhalta has gained substantial traction, with physician consideration for first-line use doubling year-over-year and second-line consideration increasing by one-third. Among recent prescriptions, Fabhalta surpasses Empaveli, Voydeya, and PiaSky, solidifying its foothold in the market.
The upcoming introduction of biosimilar eculizumab is also expected to create significant market disruption as hematologists project up to one-half of their Soliris patients may be on biosimilar eculizumab (either Bkemv (Amgen) or Epysqli (Samsung Bioepis)) by the end of 2025. Indeed, hematologists expect biosimilars’ arrival may in fact grow the overall treatment pool for PNH patients and have a ripple effect on other branded therapies as patients advance beyond C5 inhibitors. While many indicate that they will not strongly resist payer-mandated switches, some indicate they are considering self-administered options such as Empaveli or Fabhalta more strongly as a way to sidestep the complexities associated with biosimilars.
As the market continues to shift, company influence and promotional efforts remain key factors. While Alexion continues to be viewed as the most respected industry partner, Novartis has made notable gains in awareness and influence, now ranking as the second-most impactful company in physician prescribing decisions.
Looking forward, there is growing enthusiasm for Regeneron’s investigational therapy pozelimab/cemdisiran, with physician awareness surging tenfold year-over-year. Doctors view this potential treatment as a significant advance over existing options, and a majority expect to prescribe it within six months of approval. Nearly half of PNH patients are considered potential candidates for the therapy, positioning it as a likely disruptor to Ultomiris and Soliris upon its introduction.
This latest research underscores Spherix’s commitment to the PNH space, pairing with the release of its first annual chart audit study in hematology, Patient Chart Dynamix™ : Paroxysmal Nocturnal Hemoglobinuria (US), in late 2024. This study analyzed 211 PNH patient charts gathered from 88 U.S.-based hematologists and offers a detailed look at current treatment patterns, uncovering critical gaps in care, and highlighting opportunities for therapies.
With PNH treatment options expanding and biosimilars entering the market, understanding these dynamics is more crucial than ever. Through independent, data-driven research, Spherix provides invaluable strategic intelligence to help pharmaceutical manufacturers navigate the complexities of the PNH market, uncover emerging opportunities, and address persistent challenges in patient care.
RealTime Dynamix™ is an independent service providing strategic guidance through quarterly or semiannual reports, which include market trending and a fresh infusion of event-driven and variable content with each wave. The reports provide an unbiased view of the competitive landscape within rapidly evolving specialty markets, fueled by robust HCP primary research and our in-house team of experts.
Patient Chart Dynamix™ is an independent, data-driven service unveiling real patient management patterns through rigorous analysis of large-scale patient chart audits. Insights reveal the “why” behind treatment decisions, include year over year trending to quantify key aspects of market evolution, and integrate specialists’ attitudinal & demographic data to highlight differences between stated and actual treatment patterns.
About Spherix Global Insights
Spherix is a leading independent market intelligence and advisory firm that delivers commercial value to the global life sciences industry, across the brand lifecycle.
The seasoned team of Spherix experts provides an unbiased and holistic view of the landscape within rapidly evolving specialty markets, including dermatology, gastroenterology, rheumatology, nephrology, neurology, ophthalmology, and hematology. Spherix clients stay ahead of the curve with the perspective of the extensive Spherix Physician Community.
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NOTICE: All company, brand or product names in this press release are trademarks of their respective holders. The findings and opinions expressed within are based on Spherix Global Insight’s analysis and do not imply a relationship with or endorsement of the companies or brands mentioned in this press release.
Sources:
- https://www.cancer.columbia.edu/cancer-types-care/types/rare-blood-disorders/conditions/paroxysmal-nocturnal-hemoglobinuria-pnh
- https://my.clevelandclinic.org/health/diseases/22871-paroxysmal-nocturnal-hemoglobinuria
Sarah Hendry, Hematology Franchise Head Spherix Global Insights 4848794284 sarah.hendry@spherixglobalinsights.com
