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Ravulizumab

Phase 3

Paroxysmal Nocturnal Hemoglobinuria | Small molecule | Hematology |Regeneron Pharmaceuticals, Inc.|Last Updated: Apr 30, 2026

Target and mechanism

Molecular targetC5
Target classInhibitor
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

RandomizedCONTROLLEDDMC
Total Trials1
Total Enrollment202

FDA Designations

No designations recorded

Clinical trial landscape

Ravulizumab · 1 trial · 1 indication

Phase 3 1
NCT05133531A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor TreatmentParoxysmal Nocturnal Hemoglobinuria
ACTIVE NOT_RECRUITING202 Analytics
PHASE3ACTIVE NOT_RECRUITING
A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment
Paroxysmal Nocturnal HemoglobinuriaUnlock trial analytics

Study Endpoints

Primary Endpoints

Percent change in lactate dehydrogenase (LDH)
From baseline to week 26

Cohort A

Transfusion avoidance
From post-baseline day 1 through week 26

Cohort B Not requiring a red blood cell (RBC) transfusion per the protocol

Adequate control of hemolysis
From week 8 through week 26, inclusive

Cohort B LDH ≤1.5 × ULN at each visit

Secondary Endpoints

Maintenance of adequate control of hemolysis
From week 8 through week 26, inclusive
Breakthrough hemolysis
From post-baseline day 1 through week 26
Adequate control of hemolysis
From week 8 through week 26, inclusive
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Study Design & Arms

AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Cohort AEXPERIMENTALRandomized 1:1
Cohort BEXPERIMENTALRandomized 1:1

Interventions

NameTypeDescription
RavulizumabDRUGAdministered Intravenous (IV) per the protocol
PozelimabDRUGAdministered IV and subcutaneous (SC) per the protocol
CemdisiranDRUGAdministered SC per the protocol
EculizumabDRUGAdministered IV per the protocol
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites68

Key Inclusion Criteria: 1. Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNH granulocytes or monocytes as described in the protocol 2. Active disease, as defined by the presence of 1 or more PNH-related signs or symptoms as described in the protocol 3. LDH level ≥2 × UL...

Countries:United StatesBrazilCanadaChinaColombiaGreeceHungaryIndiaItalyJapanJordanMalaysiaMexicoPeruPhilippinesPolandRomaniaSingaporeSouth KoreaSpainTaiwanThailandTurkey (Türkiye)United Kingdom
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Frequently asked questions about Ravulizumab

What is Ravulizumab used for?

Ravulizumab is used for Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare blood disorder. It is being studied in adult patients with PNH who have not recently received or have not received complement inhibitor treatment. The drug is currently in Phase 3 clinical development for this indication.

Who makes Ravulizumab?

Ravulizumab is being developed by Regeneron Pharmaceuticals, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol REGN. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with Paroxysmal Nocturnal Hemoglobinuria.

What phase is Ravulizumab in?

Ravulizumab is in Phase 3 clinical development. It is being evaluated in an active, not recruiting Phase 3 study for Paroxysmal Nocturnal Hemoglobinuria. The drug is investigational and has not been approved by regulatory authorities based on the available information.

What clinical trials is Ravulizumab in?

Ravulizumab is being studied in a Phase 3 clinical trial with the identifier NCT05133531. This trial is evaluating the safety and efficacy of the drug in adult patients with Paroxysmal Nocturnal Hemoglobinuria who have not recently received or have not received complement inhibitor treatment. The trial is active but not recruiting.

How does Ravulizumab work?

Ravulizumab is a monoclonal antibody, as indicated by its target class of -mab. It is being studied for its effects in Paroxysmal Nocturnal Hemoglobinuria, a condition involving complement system dysregulation. The specific molecular target of Ravulizumab has not been disclosed in the available information.

Is Ravulizumab the same as Pozelimab or Cemdisiran?

Ravulizumab is not the same as Pozelimab or Cemdisiran. The Phase 3 clinical trial NCT05133531 is evaluating a combination therapy of Pozelimab plus Cemdisiran in patients with Paroxysmal Nocturnal Hemoglobinuria. Ravulizumab is a separate drug being developed by Regeneron Pharmaceuticals for the same condition.