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Last reviewed 2026-06-10 · How we verify

Congenital hemolytic uremic syndrome — Treatment Landscape & Competitive Intelligence

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

Congenital hemolytic uremic syndrome (disease) competitive landscape: 2 marketed treatments tracked, 0 Phase 3 candidates, 0 Phase 2 candidates. Recent regulatory actions and upcoming PDUFA dates across the entire treatment set.

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

Marketed treatment landscape

Approved drugs treating Congenital hemolytic uremic syndrome, deduplicated by molecule. See the full disease page for line-of-therapy detail.

Drug	Generic	Sponsor	Class	Target	Line of therapy	First approval
H5G1.1	ECULIZUMAB	AstraZeneca	Complement Inhibitor [EPC]	C5		2007-01-01
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

Apellis Pharmaceuticals, Inc. · 1 drug in Congenital hemolytic uremic syndrome
AstraZeneca · 1 drug in Congenital hemolytic uremic syndrome

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Every new regulatory action, PDUFA date, or trial completion in Congenital hemolytic uremic syndrome:

Cite this brief

Drug Landscape (2026). Congenital hemolytic uremic syndrome — Treatment Landscape & Competitive Intelligence Brief. https://druglandscape.com/ci/disease/congenital-hemolytic-uremic-syndrome. Accessed 2026-06-10.

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