PNH's 800-1,200 patient EVH subpopulation, not the full 15,000-20,000 prevalence estimate, defines the complement-inhibitor era opportunity.

PNH is a clonal haematopoietic stem cell disorder caused by somatic PIG-A mutation, leading to GPI-anchor deficiency and complement-mediated red blood cell lysis. US prevalence is estimated at 15,000 to 20,000 patients, median age 35, with equal sex distribution. The disease spans three clinically and commercially distinct axes, haemolysis-dominant, aplasia-dominant, and thrombotic PNH, each requiring different treatment approaches and commercial micro-segmentation rather than a single undifferentiated patient count.

Mean diagnostic delay is 2.4 years, driven by misdiagnosis as autoimmune haemolytic anaemia, aplastic anaemia, or MDS. Approximately 15 to 20% of aplastic anaemia patients harbour PNH clones, forming the largest undiagnosed patient pool. Anti-C5 therapy has dramatically reduced thrombotic mortality, but 800 to 1,200 patients with extra-vascular haemolysis, persistent anaemia despite IV complement inhibition, define the residual unmet need and the commercial rationale for proximal complement inhibitors such as iptacopan.

The EVH subpopulation, not total prevalence, is where the commercial case actually lives.

Five questions this report answers:

Q1 - What is the size and clinical characterisation of the EVH-dominant US PNH subpopulation?

Q2 - What is the diagnostic pathway for PNH, and where are the largest undiagnosed patient pools?

Q3 - How does the PNH disease spectrum drive different treatment decisions and commercial micro-segmentation?

Q4 - Which US PNH subpopulations represent the greatest unmet commercial need today?

Q5 - What share of aplastic anaemia patients actually harbour an undiagnosed PNH clone?

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Live report page:  https://axlrx.ai/pnh/disease-landscape/

Thanks & Regards,

Mike || Global Pharma Commercial Marketing Head

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