Market Barriers Continue to Challenge PNH Treatment Optimization Despite Emerging Options, According to Spherix Global Insights

By: Spherix Global Insights via GlobeNewswire

July 08, 2025 at 14:22 PM EDT

EXTON, PA, July 08, 2025 (GLOBE NEWSWIRE) -- Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic blood disorder that can affect individuals of any age, race, or gender, though it is most commonly diagnosed in adults in their 30s and 40s. In the United States, an estimated 400 to 500 new cases are identified annually. The clinical presentation of PNH varies widely—some patients experience only mild symptoms, while others face life-threatening complications requiring interventions such as immunosuppressive therapy or blood transfusions. ¹

Hematologists are actively adapting their prescribing strategies in paroxysmal nocturnal hemoglobinuria (PNH), with future pipeline entrants poised to further reshape the treatment landscape. According to Spherix Global Insights’ Q2 2025 update from RealTime Dynamix™: PNH (US), the availability and adoption of biosimilars are expected to significantly expand the overall treated PNH population—bringing more patients onto complement inhibitor therapy than ever before.

The Spherix research projects that biosimilar eculizumab products—Bkemv (Amgen) and Epysqli (Teva/Samsung Bioepis)—will continue to gain traction, capturing share from both branded Soliris and Ultomiris (Alexion/AstraZeneca), as hematologists grow increasingly confident in the clinical utility and cost-effectiveness of biosimilar alternatives.

Use of novel entrants in the second line is growing as well, with therapies such as Fabhalta (Novaratis) and Voydeya (AstraZeneca) capturing an increasing share of recent prescribing. These agents are selected for patients who progress or are sub-optimally controlled on C5 inhibitors. Despite these gains, access remains a major commercial hurdle.

Many patients with PNH are believed to be suboptimally managed due to persistent insurance-related barriers. Nearly all prescribers report significant challenges navigating the prior authorization process, citing inconsistent payer requirements, delays in drug procurement, and a growing reliance on artificial intelligence by insurers to further restrict coverage. This evolving dynamic has become a major source of frustration for hematologists, often delaying timely access to appropriate therapies and complicating clinical decision-making.

The competitive landscape in PNH continues to intensify, with future therapies gaining interest among both academic and community hematologists. The latest Spherix study incorporates target product profiles and prescriber expectations for emerging agents, including zaltenibart (Omeros), a MASP-3 inhibitor that has captured strong physician interest. Most hematologists anticipate zaltenibart will be adopted alongside existing first- and second-line therapies upon approval, driven by high efficacy expectations and its potential to address persistent unmet needs. Other late-stage candidates—such as pozelimab-cemdisiran (Regeneron/Alnylam) and ruxaprubart (NovelMed)—also register meaningful commercial and clinical potential among surveyed specialists.

“These results underscore that physicians aren’t just passively watching the market evolve – they’re preparing to act,” said Sarah Hendry, Hematology Franchise Head at Spherix. “With rising expectations for access support and guideline alignment, manufacturers that combine clinical value with commercial enablement will be best positioned for success.” As was seen in Paris at the IPIG international conference when Spherix presented data from their Patient Chart Dynamix™, membership in critical professional societies is growing as hematologists seek the guidance in PNH treatments and care.

The most recent release offers a comprehensive look at the U.S. PNH market, including prescribing patterns, brand dynamics, access challenges, and future projections. Delivered quarterly, RealTime Dynamix™ helps commercial teams anticipate shifts, benchmark against competitors, and support strategic planning ahead of key market events.

https://www.aamds.org/pnh

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 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. 

To learn more about Spherix Global Insights, visit spherixglobalinsights.com or connect through LinkedIn.  

For more details on Spherix’s primary market research reports and interactive dashboard offerings, visit or register here: https://clientportal.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 Insight’s analysis and do not imply a relationship with or endorsement of the companies or brands mentioned in this press release.

Sarah Hendry, Hematology Franchise Head
Spherix Global Insights
4848794284
sarah.hendry@spherixglobalinsights.com

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Market Barriers Continue to Challenge PNH Treatment Optimization Despite Emerging Options, According to Spherix Global Insights

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