Spherix Global Insights Reports Rapidly Evolving U.S. PNH Market as Ultomiris Prescriptions Reach Parity with Iron Supplements
New chart-audit data reveal faster referrals, more patients initiating advanced therapy before hematology consultation, and heightened disease awareness among community physicians.
Exton, PA, Nov. 19, 2025 (GLOBE NEWSWIRE) -- New data from Spherix Global Insights reveal that the U.S. market for paroxysmal nocturnal hemoglobinuria (PNH) is entering a new phase of dynamism, characterized by shifting referral patterns, rapid treatment adoption, and a heightened level of disease awareness among primary care physicians. According to findings from the latest Patient Chart Dynamix™: PNH service, prescriptions for Ultomiris (Alexion/AZ) are now on par with those for iron supplements, a striking indicator of how deeply advanced therapies have penetrated routine management of this rare disorder.
The audit, which evaluated 111 patient charts provided by 50 U.S. hematologists in late 2025, also revealed a meaningful improvement in referral pathways. Nearly all patients referred to hematologists in the last year came from primary care, and notably, no cases were characterized as “extremely delayed” -- a marked shift from the several such instances reported in 2024. In parallel, a growing proportion of patients are reaching hematology already initiated on advanced therapies, such as complement inhibitors, rather than relying solely on traditional supportive care. This shift indicates that community physicians are becoming more adept at identifying hallmark features of PNH, such as unexplained anemia, evidence of hemolysis, and elevated thrombosis risk, and are escalating care to specialists more promptly. Taken together, these patterns reflect a meaningful enhancement in disease recognition and management, ultimately translating into earlier intervention opportunities and improved patient outcomes.
The evolving PNH market continues to be shaped by aggressive direct-to-consumer and digital promotion, alongside heightened regulatory scrutiny of said promotion. Both Alexion/AstraZeneca and Novartis have launched major awareness campaigns for Ultomiris and Fabhalta, respectively, the latter triggering an Untitled Letter from the FDA for a ‘misleading’ storyline in its advertisement. These dynamics collectively signal an inflection point for the PNH therapeutic landscape. The near parity of Ultomiris prescriptions with iron supplements highlights the speed at which innovative therapies are reshaping patient management. Shorter referral timelines and higher rates of advanced treatment initiation prior to hematology consultation underscore growing disease awareness among community providers. Concurrently, intense promotional competition and regulatory interventions are redefining how companies can communicate benefits and risks in this rapidly evolving market.
About Patient Chart Dynamix™
Patient Chart Dynamix™ is an independent service that includes robust patient chart audits and integrated specialist surveys fielded biannually. This research provides an in-depth, real-world view of treatment practices by combining verified patient data with attitudinal insights from physicians. The series highlights clinical decision-making, treatment sequencing, and outcomes for targeted patient populations across key therapeutic areas.
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.
As a trusted advisor and industry thought leader, Spherix’s unparalleled market insights and advisory services empower clients to make better decisions and unlock opportunities for growth.
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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.
Sarah Hendry Spherix Global Insights 484-879-4284 sarah.hendry@spherixglobalinsights.com
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