VEOPOZ

(Pozelimab)

ApprovedMonoclonal antibody

Regeneron

VEOPOZ is one of 29 small molecule programs targeting Paroxysmal Nocturnal Hemoglobinuria. It is currently in Approved.

Indications

Total Trials

Active Trials

Competitors

FDA Application

NDA/BLA/ANDA regulatory filings

Pipeline

Status by Indication[3][1]

Indication

Paroxysmal Nocturnal HemoglobinuriaRecruiting

Generalized Myasthenia GravisActive, not recruiting

Geographic Atrophy (GA)Recruiting

ThrombosisRecruiting

Paroxysmal Nocturnal HemoglobinuriaPhase 3

Recruiting

Generalized Myasthenia GravisPhase 3

Active, not recruiting

Geographic Atrophy (GA)Phase 1

Recruiting

ThrombosisApproved

Recruiting

Competition

Competitive Landscape

17 competitors in Paroxysmal Nocturnal Hemoglobinuria

#DrugCompanyPhaseTrials

Soliris

AstraZeneca

Approved

25 2

Ultomiris

AstraZeneca

Approved

12 3

VEOPOZ

Regeneron

Approved

7 4

Fabhalta

Novartis

Phase 3

2 5

PIASKY

Roche

Phase 3

3 6

NTQ5082

NNanjing Chia-tai Tianqing Pharmaceutical

Phase 3

2 7

Cemdisiran

Regeneron

Phase 3

7 8

NM8074

NNovelMed Therapeutics

Phase 2

1 9

HRS-5965

CChengdu Suncadia Medicine Co., Ltd.

Phase 3

3 10

ADX-038

AADARx Pharmaceuticals, Inc.

Phase 2

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q1 2027

Phase 3Recruiting

Paroxysmal Nocturnal Hemoglobinuria

NCT05133531

Q3 2028

Phase 1Recruiting

Geographic Atrophy (GA)

NCT07230834

Q4 2028

Phase 3Recruiting

Paroxysmal Nocturnal Hemoglobinuria

NCT05744921

Phase 3Active, not recruiting

Generalized Myasthenia Gravis

NCT05070858

Q1 2030

Post-ApprovalRecruiting

Thrombosis

NCT07142343

Clinical

Trials by Indication[1]

Paroxysmal Nocturnal Hemoglobinuria7 trials

Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy

Completed· Regeneron logo

Regeneron·Phase 2·NCT04811716·24 enrolled

Compassionate Use of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria

No longer available· Regeneron logo

Regeneron··NCT06028594

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

Recruiting· Regeneron logo

Regeneron·Phase 3·NCT05133531·190 enrolled

Show 4 more trials

Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy

Completed· Regeneron logo

Regeneron·Phase 2·NCT04888507·6 enrolled

Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria

Terminated· Regeneron logo

Regeneron·Phase 3·NCT05131204·3 enrolled

An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria

Recruiting· Regeneron logo

Regeneron·Phase 3·NCT05744921·202 enrolled

A Single Arm Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Control of Intravascular Hemolysis on Currently Available C5 Inhibitor Therapy

Recruiting· Regeneron logo

Regeneron·Phase 3·NCT07154745·35 enrolled

MPA2 trials

Compassionate Use of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy

Approved for marketing· Regeneron logo

Regeneron··NCT06003881

Compassionate Use of Pozelimab

Approved for marketing· Regeneron logo

Regeneron··NCT05232110

Adult2 trials

Study of a Single Subcutaneous Dose of Pozelimab Produced From Two Different Manufacturing Processes in Healthy Adult Participants

Completed· Regeneron logo

Regeneron·Phase 1·NCT04491838·40 enrolled

Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Pozelimab in Combination With Cemdisiran in Healthy Adult Volunteers

Completed· Regeneron logo

Regeneron·Phase 1·NCT04601844·19 enrolled

Intravenous1 trial

A Study to Examine the Safety, Tolerability and Biological Effects of Single Doses of Subcutaneously and Intravenously Administered Pozelimab as Monotherapy and in Combination With Single Doses of Subcutaneously Administered Cemdisiran in Adult Japanese Healthy Volunteers

Completed· Regeneron logo

Regeneron·Phase 1·NCT04940364·30 enrolled

Thrombosis1 trial

A Study to Test the Safety of Pozelimab in Pediatric Participants 1 to 5 Years of Age With a Rare Disease Called CHAPLE (Complement Hyperactivation, Angiopathic Thrombosis, Protein-losing Enteropathy) Disease

Recruiting· Regeneron logo

Regeneron·Phase 4·NCT07142343·5 enrolled

4 more indications

Efficacy and Safety1 trial

Open-Label Efficacy and Safety Study of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy (CHAPLE Disease)

Completed· Regeneron logo

Regeneron·Phase 2/3·NCT04209634·10 enrolled

Age-related Macular Degeneration (AMD)1 trial

A Multicenter, Randomized, Double-Masked, Placebo-Controlled Phase 3 Study of the Efficacy, Safety, and Tolerability of Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration

Recruiting· Regeneron logo

Regeneron·Phase 3·NCT06541704·975 enrolled

Geographic Atrophy (GA)1 trial

A Phase 1 Dose-Escalation and Repeated-Dose Study of the Safety and Tolerability of Intravitreal Pozelimab in Participants With Geographic Atrophy

Recruiting· Regeneron logo

Regeneron·Phase 1·NCT07230834·54 enrolled

Generalized Myasthenia Gravis1 trial

Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy and Cemdisiran Monotherapy in Patients With Symptomatic Generalized Myasthenia Gravis

Active, not recruiting· Regeneron logo

Regeneron·Phase 3·NCT05070858·288 enrolled

Regulatory

FDA Approval Timeline[4]

Aug 18, 2023

VEOPOZ

BLA BLA761339

Sources

References

[1]
ClinicalTrials.gov — 17 trial records for Pozelimab. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Pozelimab compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Pozelimab. EMBL-EBI & Wellcome Sanger Institute.
[4]
OpenFDA — FDA approval records (BLA BLA761339). U.S. Food & Drug Administration.
[5]
SEC EDGAR — Regeneron company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Monoclonal antibody[2]
Highest Phase: Approved[1]
Company: Regeneron

VEOPOZ

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q1 2027

Q3 2028

Q4 2028

Q1 2030

Trials by Indication[1]

Paroxysmal Nocturnal Hemoglobinuria7 trials

MPA2 trials

Adult2 trials

Intravenous1 trial

Thrombosis1 trial

Efficacy and Safety1 trial

Age-related Macular Degeneration (AMD)1 trial

Geographic Atrophy (GA)1 trial

Generalized Myasthenia Gravis1 trial

FDA Approval Timeline[4]

References

Drug Info

Quick Facts

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