Iptacopan's orphan-drug status secured an established benefit and a substantial quality-of-life finding in Germany without a comparator dossier fight.

PNH has no confirmed German prevalence data of its own. The DGHO's Onkopedia guideline extrapolates instead from British and French registries, landing on an estimated 16 cases per million and 1.3 new diagnoses per million annually, and states plainly that Germany-specific figures do not exist. Diagnosis runs on GPI-anchor flow cytometry: at least two GPI-anchored markers must show deficient or reduced expression across at least two cell lineages. Two centres anchor the referral network: the German PNH-Register 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.

Iptacopan's route to reimbursement skipped the standard AMNOG fight entirely. As an orphan-designated therapy, its additional benefit counted as established through EMA approval, so IQWiG never had to build a head-to-head dossier against anti-C5 therapy. The G-BA went further on 19 December 2024, finding a substantial additional benefit specifically on quality of life for patients switching from anti-C5 therapy, and in March 2025 declined to require accompanying data collection. Crovalimab has no such shortcut: its dossier, submitted 12 September 2024, remains under active IQWiG assessment. Ravulizumab's only Germany-specific finding found no additional benefit against eculizumab.

Future anti-C5 entrants face the comparator dossier fight iptacopan skipped entirely.

Five questions this report answers:

Q1 - What does the orphan pathway actually establish, and what evidentiary bar did iptacopan skip?

Q2 - Where does German PNH referral concentrate, and how is the addressable population sized without a confirmed national count?

Q3 - What is crovalimab's AMNOG status today, and what does ravulizumab's 2022 pediatric rejection signal?

Q4 - How does Germany's AMNOG/G-BA framework shape PNH market access for future anti-C5 entrants?

Q5 - Why did the G-BA decline to require a parallel data-collection registry for iptacopan?

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Live report page:  https://axlrx.ai/pnh/germany/competitive-intelligence/

Thanks & Regards,

Mike || Global Pharma Commercial Marketing Head

Email-      hello@axlrx.ai

Web-      https://axlrx.ai/