UK PNH diagnosis takes just 6-12 months through Leeds, yet 30% of the ~600 tracked patients also have aplastic anaemia.

PNH is a clonal haematopoietic stem cell disorder caused by somatic PIG-A mutation, producing GPI-anchor deficiency and complement-mediated red blood cell lysis. The Leeds National PNH Service runs the UK's single national registry: approximately 600 patients on complement inhibitor therapy, plus 100 to 150 additional patients with small PNH clones monitored without treatment. Annual new UK diagnoses run 50 to 70, with a median age at diagnosis of 38 to 42 years and an equal gender distribution across the tracked cohort.

UK PNH survival on complement inhibitor therapy now approaches that of the general population, a landmark outcome for a historically fatal disease. Around 30% of UK PNH patients have concurrent aplastic anaemia features requiring dual management: complement inhibitor alongside immunosuppressive therapy, eltrombopag, or a bone marrow transplant discussion, coordinated through the Leeds multidisciplinary service and 15 JACIE-accredited NHS BMT centres across the country.

Nearly a third of UK PNH patients need dual disease management.

Five questions this report answers:

Q1 - What is the size of the NHS-commissioned PNH population against the pending iptacopan decision?

Q2 - Why does the UK achieve a materially shorter PNH time-to-diagnosis than other markets?

Q3 - How does the 30% PNH-aplasia overlap population get managed within the NHS?

Q4 - What does the pending iptacopan NICE decision mean for UK PNH access?

Q5 - How many UK PNH patients are monitored on the Leeds registry without active treatment?

Share your commercial question with us. We'll align on scope — then build the right intelligence around it.

→ moatrx.com/axlrx.html

#PNH #NHS #NICE #RareDisease #UKHealthcare #DiseaseLandscape

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

Thanks & Regards,

Mike || Global Pharma Commercial Marketing Head

Email-                    hello@axlrx.ai

Web-                    https://axlrx.ai/