PNH Market in Transition as Second-Line Therapy Opportunities Grow
Event summary
- Spherix Global Insights' 2026 report reveals 30% of PNH patients not achieving adequate hemoglobin response on current therapy.
- Only 24% of PNH patients are currently treated in the second line or later, highlighting a significant opportunity for advanced therapies.
- 61% of hematologists report changing their PNH management approach in the past year, primarily by increasing use of newer agents.
- More than half of physicians are prescribing biosimilar eculizumab, anticipating it could represent roughly half of eculizumab-treated patients by year-end 2026.
The big picture
The PNH market is undergoing a significant transition with the expansion of second-line therapy opportunities. Despite the introduction of newer agents, persistent barriers such as clinical practice gaps, referral pathway issues, and payer access challenges are tempering their adoption. The evolving therapeutic landscape, including the rise of biosimilars, is reshaping the market dynamics and prescribing behaviors, reflecting the continued unmet needs within the PNH patient population.
What we're watching
- Therapy Adoption
- How the adoption of newer agents like Fabhalta, Voydeya, and PiaSky will evolve amid ongoing insurance and access hurdles.
- Biosimilar Impact
- The pace at which biosimilars will reshape payer dynamics and prescribing behaviors in the PNH market.
- Pipeline Strategies
- Whether combination regimens and proximal complement pathway approaches will address the unmet needs of the growing 2L+ patient population.
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