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Last reviewed 2026-06-10 · How we verify

Paroxysmal nocturnal hemoglobinuria — Treatment Landscape & Competitive Intelligence

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Paroxysmal nocturnal hemoglobinuria (disease) competitive landscape: 6 marketed treatments tracked, 0 Phase 3 candidates, 0 Phase 2 candidates. Recent regulatory actions and upcoming PDUFA dates across the entire treatment set.

6 marketed 0 Phase 3 0 Phase 2 Live · 30-min refresh

Marketed treatment landscape

Approved drugs treating Paroxysmal nocturnal hemoglobinuria, deduplicated by molecule. See the full disease page for line-of-therapy detail.

Drug	Generic	Sponsor	Class	Target	First approval
Fabhalta	IPTACOPAN	Novartis		Factor B	2023-01-01
Ultomiris	RAVULIZUMAB	AstraZeneca	Complement Inhibitor [EPC]	Complement C5	2018-01-01
H5G1.1	ECULIZUMAB	AstraZeneca	Complement Inhibitor [EPC]	C5	2007-01-01
ULTOMIRIS	RAVULIZUMAB-CWVZ	ALEXION PHARM		Complement C5
Ultomiris	Ultomiris	Alexion Pharmaceuticals, Inc.		Complement C5
Soliris	Soliris	Apellis Pharmaceuticals, Inc.		Complement C5

Phase 3 pipeline

No Phase 3 pipeline candidates tracked.

Phase 2 pipeline

No Phase 2 pipeline candidates tracked.

Recent regulatory actions (last 90 days)

No regulatory actions in the last 90 days for this treatment set.

Upcoming PDUFA dates (next 180 days)

No PDUFA dates in the next 180 days for this treatment set.

Sponsor landscape

AstraZeneca · 2 drugs in Paroxysmal nocturnal hemoglobinuria
ALEXION PHARM · 1 drug in Paroxysmal nocturnal hemoglobinuria
Alexion Pharmaceuticals, Inc. · 1 drug in Paroxysmal nocturnal hemoglobinuria
Apellis Pharmaceuticals, Inc. · 1 drug in Paroxysmal nocturnal hemoglobinuria
Novartis · 1 drug in Paroxysmal nocturnal hemoglobinuria

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Cite this brief

Drug Landscape (2026). Paroxysmal nocturnal hemoglobinuria — Treatment Landscape & Competitive Intelligence Brief. https://druglandscape.com/ci/disease/paroxysmal-nocturnal-hemoglobinuria. Accessed 2026-06-10.

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Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing