Paroxysmal Nocturnal Hemoglobinuria – Unmet Need – Unmet Need – Paroxysomal Nocturnal Hemoglobinuria (US/EU)
The treatment landscape for paroxysmal nocturnal hemoglobinuria (PNH) has shifted dramatically in the past decade, moving beyond supportive care to transformative complement inhibition therapies. Alexion’s eculizumab (Soliris) and ravulizumab (Ultomiris), alongside Apellis’s pegcetacoplan (Empaveli), set new standards for improved survival and quality of life. More recently, Novartis’s iptacopan (Fabhalta) became the first FDA- and EC-approved oral therapy, followed by AstraZeneca’s danicopan, an add-on therapy (Voydeya), expanding patient choice. Roche’s crovalimab (PiaSky), approved in 2024, introduced the first once-monthly SC option for both adult and pediatric patients, underscoring the pace of innovation. Late-stage candidates such as Regeneron and Alnylam’s cemdisiran-pozelimab, the first siRNA-based approach targeting the terminal complement pathway, highlight the next wave of disruption. With multiple modalities now competing for prescriber adoption, understanding clinical decision drivers and positioning opportunities is critical. The PNH market is evolving rapidly, and staying informed on these developments is essential for identifying differentiation levers and future growth.
Questions answered
- What are the key treatment drivers and goals in PNH treatment? How well do current therapies perform on these goals?
- What drug attributes are key influences, which have limited impact, and which are hidden opportunities?
- Where do surveyed hematologists perceive the largest gaps in the treatment of PNH?
- What trade-offs across different clinical attributes and prices are acceptable to U.S. and European hematologists for a hypothetical new PNH drug?
Markets covered: United States, United Kingdom, France, Germany
Primary research: Survey of 60 U.S. and 31 European hematologists, fielded in December 2025
Key companies: AstraZeneca, Roche, Apellis Pharmaceuticals / Sobi, Novartis
Key drugs: Ultomiris, Piasky, Empaveli / Aspaveli, Fabhalta, Voydeya
Product description
Unmet Need offers insight into key treatment drivers and goals, the performance of current therapies, and the remaining commercial opportunities, enabling you to:
- Evaluate clinical and nonclinical product attributes that influence treatment decisions through physicians’ weightings and analysis of stated vs. derived importance.
- Pinpoint areas of high unmet need by assessing current drug performance against key attributes and treatment drivers.
- Analyze market scenarios for different target product profiles using the TPP Simulator.
Key feature
Target Product Profile (TPP) simulator tool allows for customizable market simulations based on conjoint analysis. Compare up to seven TPPs across multiple disease-specific attributes and price points to gauge which variables influence prescribing behavior.
