DE PNH Disease Landscape

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. Diagnosis requires GPI-anchor flow cytometry showing deficient or reduced expression of at least two GPI-anchored markers across at least two cell lineages, typically granulocytes and reticulocytes. In Germany, that testing and the resulting patient population concentrate almost entirely at two institutions: the German PNH-Register, housed at Ulm's Institute for Clinical Transfusion Medicine and Immunogenetics, and the West German Cancer Center at University Hospital Essen. Both feed patients into the International PNH Registry, the same dataset underpinning the long-term ravulizumab and eculizumab outcomes literature.

The DGHO's own Onkopedia clinical guideline, the reference document German haematologists use, states directly that Germany-specific prevalence and incidence figures do not exist. In their place, it extrapolates an estimated 16 cases per million and 1.3 new diagnoses per million annually from British and French registry data. That borrowed-estimate approach has a direct commercial consequence: any epidemiology-based market sizing for a new PNH therapy launching in Germany inherits the uncertainty of a foreign-registry extrapolation rather than a domestic count, and the two-centre referral concentration means patient-finding runs through a narrower institutional funnel than in markets with broader registry infrastructure.

Every AXLRx assessment is built from primary sources (national clinical guidelines, referral-centre registries, and peer-reviewed literature), not secondary summaries. Findings are independently verified before inclusion.

PNH Germany Disease Landscape sources: DGHO Onkopedia PNH clinical guideline (German epidemiology statement and borrowed estimates); German PNH-Register (Ulm); West German Cancer Center (Essen); International PNH Registry.