Paroxysmal nocturnal hemoglobinuria

PNH · Rare Disease · 5 drugs · 3 indications

Complement-mediated hemolytic anemia.

Competitive Landscape (5 drugs)

Drug	Company	Mechanism	Modality	Route	Stage
Ultomiris	AZN	C5 inhibitor	Antibody	IV	APPROVED
Soliris	AZN	C5 inhibitor	Antibody	IV	APPROVED
Fabhalta	NVS	Complement factor B inhibitor	Small molecule	Oral	APPROVED
Veopoz	REGN	C5 inhibitor	Monoclonal antibody	SC	APPROVED
Aspaveli	SOBI	C3 complement inhibitor	PEGylated peptide	SC	APPROVED

Indications (3)

PNH

Ultomiris APPROVEDSoliris APPROVEDAspaveli APPROVED

PNH (complement inhibitor-naive)

Fabhalta APPROVED

Paroxysmal nocturnal hemoglobinuria (C5 combo)

Veopoz PHASE3

Upcoming Catalysts

Ultomiris - IgAN - Ph3 - Topline (ICAN)CLINICAL

AZNH1 2026

Ultomiris - HSCT-TMA - Ph3 - ToplineCLINICAL

AZNH1 2026

Pozelimab+Cemdisiran - gMG - Ph3 Topline (NIMBLE)CLINICAL

REGN2026-2027

Fabhalta - IgAN - Full Approval DataCLINICAL

NVSH2 2026

Data from Supabase · Updated 2026-03-24