Exton, PA, March 05, 2026 (GLOBE NEWSWIRE) -- Spherix Global Insights today announces the release of its latest Market Dynamix™: Paroxysmal Nocturnal Hemoglobinuria (PNH) (US) 2026 study, revealing a sizable population of patients eligible for advanced second-line and later (2L+) therapy – while highlighting persistent clinical practice, referral pathway, and payer access barriers that continue to temper adoption of recently approved agents.
Based on a survey of 71 U.S. hematologists and in-depth qualitative interviews fielded in early 2026, the report captures a PNH market in transition. While treatment innovation in PNH has accelerated over the past two years, nearly one in four patients remain sub-optimally managed, and approximately 30% are not achieving what physicians consider an adequate hemoglobin response on current therapy, reflecting ongoing residual anemia and breakthrough hemolysis in real-world practice.
Despite expanded therapeutic options, including C5 inhibitors, C3 inhibitors, and emerging proximal pathway agents, only 24% of PNH patients are currently treated in the second line or later, and 22% of patients are considered not optimally managed. This gap underscores a meaningful opportunity for advanced therapies to address persistent anemia, breakthrough hemolysis, and quality-of-life limitations.
Hematologists report that preventing and managing breakthrough hemolysis, navigating insurance barriers, and improving overall efficacy remain among the top challenges in PNH care. Referral timing further compounds these issues, with a sizeable proportion of patients referred to specialized centers later than physicians would prefer.
Although familiarity with newer agents, including Fabhalta (iptacopan, Novartis), Voydeya (danicopan, Alexion), and PiaSky (crovalimab, Roche), is high, adoption remains uneven. Non-use is largely driven by lack of familiarity (particularly for PiaSky), concerns about the depth of long-term clinical data, waiting for the “right” patient, and ongoing insurance or access hurdles.
This hesitation persists even as 61% of hematologists report making changes to their PNH management approach in the past year, most commonly increasing use of newer agents and longer-acting therapies.
The evolving PNH landscape now includes C5 complement inhibitors, C3 inhibitors, oral proximal complement pathway therapies, factor inhibitors, and emerging biosimilars. Physicians report diverging opinions on optimal sequencing, particularly regarding when to initiate or transition from C5 inhibition, how to manage residual anemia, and when to escalate therapy in patients with ongoing hemolysis.
At the same time, biosimilars are reshaping payer dynamics. More than half of physicians report prescribing biosimilar eculizumab, citing cost savings and formulary access advantages, with many anticipating biosimilars could represent roughly half of eculizumab-treated patients by year-end 2026.
As the therapeutic bar rises in PNH, physicians increasingly prioritize sustained efficacy, improvement in overall survival, reduction in symptom burden, and thrombosis risk mitigation when evaluating new agents. Pipeline strategies, including combination regimens and proximal complement pathway approaches, are generating meaningful interest, reflecting continued unmet need within the growing 2L+ and sub-optimally controlled patient population.
About Market Dynamix™
Market Dynamix™ is Spherix Global Insights’ independent service analyzing specialty markets expected to undergo paradigm shifts within three to five years. The PNH 2026 report includes quantitative and qualitative insights into current treatment paradigms, unmet need, market potential, prescribing intent, and the anticipated impact of pipeline agents.
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 market access, dermatology, gastroenterology, rheumatology, nephrology, neurology, ophthalmology, hematology, and oncology. Spherix clients stay ahead of the curve with the perspective of the extensive Spherix Physician Community.
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Spherix Global Insights Contacts
Sarah Hendry, Hematology/Oncology Franchise Head
Sarah.hendry@spherixglobalinsights.com
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 Insights’ analysis and do not imply a relationship with or endorsement of the companies or brands mentioned in this press release.
Sarah Hendry, Hematology/Oncology Franchise Head Spherix Global Insights 484-879-4284 sarah.hendry@spherixglobalinights.com
