The MONDO definition describes PNH as an acquired clonal hematopoietic stem cell disorder. No causative genes and no inheritance pattern are certified in this packet, so no genetic cause or mode of inheritance is described here. The packet's synonym list contains both acquired and hereditary or inherited labels, and two subtype entries, paroxysmal nocturnal hemoglobinuria 1 and paroxysmal nocturnal hemoglobinuria 2, are recorded.

Diagnostic methods, diagnostic criteria, and newborn screening information are not certified in this packet, so no diagnostic approach is described here.

The FDA records in this packet list, with ACTIVE market status, EPYSQLI (eculizumab-aagh), BKEMV (eculizumab-aeeb), SOLIRIS (eculizumab), ULTOMIRIS (ravulizumab), PIASKY (crovalimab), VOYDEYA (danicopan), FABHALTA (iptacopan), and EMPAVELI (pegcetacoplan). The orphan drug records list Soliris, Ultomiris, Piasky, Voydeya, Fabhalta, and Empaveli as APPROVED for treatment of PNH. A further FDA entry, SYFOVRE (pegcetacoplan), has no indication stated in the packet and is not described here as a PNH treatment. Several additional agents, including cemdisiran, pozelimab, and nomacopan, hold orphan drug designation for PNH, which is a designation and not an approval, and two designation records are marked WITHDRAWN. Foundational therapies and GeneReviews management text are not certified in this packet.

Prognosis is not certified in this packet; no natural history or outcome source is supplied.

ClinicalTrials.gov records in the packet include NCT05744921, a Phase 3 long-term study of pozelimab plus cemdisiran combination therapy in adults sponsored by Regeneron Pharmaceuticals (recruiting; 2023-03-07 to 2028-10-10). NCT04654468 is a Phase 3 study of crovalimab in participants not previously treated with complement inhibition, sponsored by Hoffmann-La Roche (active, not recruiting; 2021-03-17 to 2027-01-15). NCT06934967 is a Phase 3 study of iptacopan in pediatric participants sponsored by Novartis Pharmaceuticals (recruiting; 2025-10-28 to 2031-11-13). Active clinical trials for this condition are listed on ClinicalTrials.gov.