VivoSeq
Hematology

Atypical Hemolytic-Uremic Syndrome

3

Pipeline Programs↗

5

Active Trials↗

4

Companies Involved↗

Atypical Hemolytic-Uremic Syndrome is an early-stage landscape with 3 programs across 4 companies. The most active players are AstraZeneca, Novartis, Roche. 1 therapy is approved with 1 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

28/100

Pipeline Velocity

6/100

Mech. Concentration

67/100

Completion Rate

78/100

Attrition Rate

14/100

Competition

Competitive Landscape

Bubble size = clinical trials

Approved
Phase 3
Phase 2
Phase 1
Preclinical

Key Players

Top Companies Companies ranked by the number of pipeline programs they run for this disease.

AstraZeneca logo

AstraZeneca

2 programs

Phase 3Novartis logo

Novartis

1 program

ApprovedRoche logo

Roche

0 programs

PreclinicalAlnylam Pharmaceuticals logo

Alnylam Pharmaceuticals

0 programs

Preclinical

Pipeline

Programs by Phase

Preclinical1

Eculizumab

AstraZeneca

Phase 1
No programs
Phase 2
No programs
Phase 31

Ravulizumab

AstraZeneca

Approved1

Iptacopan

Novartis

Analysis

Modality Breakdown

67%

Monoclonal antibody

2 of 3 classified programs — the dominant modality here.

33%ALL OTHERSSmall molecule133%

Monoclonal antibody leads; the ring shows all 3 classified programs, each share of the total. Modality classified for 100% of 3 pipeline programs.

Analysis

Trial Status

14

clinical trials

Completed7 · 50%
Active, not recruiting3 · 21%
Recruiting2 · 14%
Withdrawn1 · 7%
Terminated1 · 7%

Clinical Trials

Trial Activity

14 trials

Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
Recruiting·Novartis logoNovartis·Phase 3·NCT05935215·50 enrolled
A Non-interventional Study Evaluating Ravulizumab Treatment Outcomes in Polish Patients With Atypical Hemolytic Uremic Syndrome
Recruiting·AstraZeneca logoAstraZeneca·N/A·NCT07399730·80 enrolled
A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Pediatric Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
Active, not recruiting·Roche logoRoche·Phase 3·NCT04958265·41 enrolled
Efficacy and Safety of Iptacopan (LNP023) in Adult Patients With Atypical Hemolytic Uremic Syndrome Naive to Complement Inhibitor Therapy
Active, not recruiting·Novartis logoNovartis·Phase 3·NCT04889430·34 enrolled
A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Adult and Adolescent Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
Active, not recruiting·Roche logoRoche·Phase 3·NCT04861259·83 enrolled
An Open-label, Multi-center Clinical Trial of Eculizumab in Adult Patients With Atypical Hemolytic-uremic Syndrome
Completed·AstraZeneca logoAstraZeneca·Phase 2·NCT01194973·44 enrolled
An Open-Label, Multi-Center Clinical Trial of Eculizumab in Pediatric Patients With Atypical Hemolytic-Uremic Syndrome
Completed·AstraZeneca logoAstraZeneca·Phase 2·NCT01193348·22 enrolled
Study of ALXN1210 in Complement Inhibitor Treatment-Naïve Adult and Adolescent Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
Completed·AstraZeneca logoAstraZeneca·Phase 3·NCT02949128·58 enrolled
Open Label Controlled Trial of Eculizumab in Adult Patients With Plasma Therapy-sensitive Atypical Hemolytic Uremic Syndrome (aHUS)
Completed·AstraZeneca logoAstraZeneca·Phase 2·NCT00838513·15 enrolled
Study of Ravulizumab in Children and Adolescents With Atypical Hemolytic Uremic Syndrome (aHUS)
Completed·AstraZeneca logoAstraZeneca·Phase 3·NCT03131219·34 enrolled
Eculizumab in Pediatric and Adult Participants With Atypical Hemolytic Uremic Syndrome (aHUS) in China
Completed·AstraZeneca logoAstraZeneca·Phase 3·NCT05876351·25 enrolled
The Safety and Efficacy of Eculizumab in Japanese Patients With Atypical Hemolytic Uremic Syndrome (aHUS)
Completed·AstraZeneca logoAstraZeneca·Phase 2·NCT01757431·2 enrolled
Safety and Efficacy Study of OMS721 in Patients With Atypical Hemolytic Uremic Syndrome
Terminated·OOmeros Corporation·Phase 3·NCT03205995·6 enrolled
A Study of an Investigational Drug, Cemdisiran (ALN-CC5), in Patients With Atypical Hemolytic Uremic Syndrome
Withdrawn·Alnylam Pharmaceuticals logoAlnylam Pharmaceuticals·Phase 2·NCT03303313

Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)