> Trialight public data. Index of pages: https://trialight.com/llms.txt > Web page: https://trialight.com/data/asset/pozelimab-b1dde3e4-bc26-40a5-98bd-c8a34129af59 > Record changed: 2026-11-04 # Pozelimab Highest phase reached Approved Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4298086) Drug type Antibody Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4298086) First approval year 2023 Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4298086) ## Development status **First approval year** 2023 Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4298086) **Listing at source** Listed **Highest phase reached** Approved Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4298086) ## Names and chemistry **Drug type** Antibody Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4298086) **Drug name** Pozelimab Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4298086) ### Brand names (2) - veopoz - Veopoz ### Other names (20) - pozelimab - Pozelimab - pozelimab \[INN\] - Pozelimab \[MI\] - POZELIMAB \[USAN\] - Pozelimab \[WHO-DD\] - pozelimab bbfg - Pozelimab bbfg \[WHO-DD\] - pozelimab bbfg who dd - pozelimab cemdisiran - pozelimab inn - pozelimab mi - pozelimab usan - pozelimab who dd - Pozelimab-bbfg - regn 3918 - REGN-3918 - regn3918 - Regn3918 - REGN3918 ## Identifiers **ATC classification codes** - L04AJ11 Sources for recorded values Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4298086) **CAS registry numbers** - 2096328-94-6 **ChEMBL ID** CHEMBL4298086 Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4298086) **FDA ingredient codes (UNII)** - 0JJ21K6L2I ### RxNorm IDs (RxCUI) (2) - 2663938 - 2663939 ## Regulatory information ### United States **Applicant** REGENERON PHARMACEUTICALS **Approval date** 18 Aug 2023 **Application number** BLA761339 ### Other regulatory facts **Patents** 0 **Designations** 4 **Earliest recorded loss of exclusivity** 18 Aug 2030 **First in class** Yes **Fast track** Yes **Breakthrough designation** No **Orphan designation** No **Priority review** Yes **Orphan exclusivity** No **Accelerated approval** No **Availability** Prescription only **Boxed warning** Yes ## Approval status **United States (FDA)** Approved ## Pharmacology ### Drug classification **Pharmacology name** pozelimab **Type** Antibody **Drug name stem** \-mab **Approval recorded by Guide to Pharmacology** Yes #### Administration routes - Parenteral ### Mechanisms of action (2) - **Action** Binding **Action type** Binding **Target** complement C5 **Gene symbol** complement C5 **Median affinity** 9.59 **Affinity units** pKd - **Mechanism** Complement C5 inhibitor **Action type** Inhibitor **Target** Complement C5 **Protein identifier** P01031 ### Open Targets mechanisms - **Mechanism** Complement C5 inhibitor **Action type** Inhibitor **Target** Complement C5 ### Investigated indications (4) - **Condition** Myasthenia Gravis **Highest recorded phase** 3 **Condition identifier** EFO:0004991 **Subject identifier** D009157 - **Condition** Protein-Losing Enteropathies **Highest recorded phase** 4 **Condition identifier** HP:0002243 **Subject identifier** D011504 - **Condition** Hemoglobinuria **Highest recorded phase** 3 **Condition identifier** MONDO:0100244 **Subject identifier** D006456 - **Condition** Hemoglobinuria, Paroxysmal **Highest recorded phase** 1 **Condition identifier** MONDO:0100245 **Subject identifier** D006457 ### Open Targets indications (8) - **Condition identifier** EFO\_0004991 **Highest recorded phase** 3 - **Condition identifier** EFO\_1001492 **Highest recorded phase** 3 - **Condition identifier** HP\_0002243 - **Condition identifier** HP\_0002243 **Highest recorded phase** 4 - **Condition identifier** MONDO\_0005150 **Highest recorded phase** 3 - **Condition identifier** MONDO\_0007827 **Highest recorded phase** 1 - **Condition identifier** MONDO\_0009688 **Highest recorded phase** 3 - **Condition identifier** MONDO\_0100244 **Highest recorded phase** 3 ### Warnings (2) - **Warning** Black Box Warning **Effect** infectious disease - **Warning** Black Box Warning **Effect** neurotoxicity ### Open Targets warnings (2) - **Warning** Black Box Warning **Effect** infectious disease **Country** United States - **Warning** Black Box Warning **Effect** neurotoxicity **Country** United States ### Trial references (9) - NCT03946748 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT03946748) - NCT04162470 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT04162470) - NCT04811716 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT04811716) - NCT04888507 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT04888507) - NCT05131204 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05131204) - NCT05133531 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05133531) - NCT05744921 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05744921) - NCT04209634 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT04209634) - NCT05070858 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05070858) ## Dosing **Dose** 30 mg/kg **Route** Intravenous **How often** Once weekly starting on Day 8 ## Chemical properties **Prodrug** No **Chirality** Single enantiomer **Inorganic** No **Natural product** No ### Sequences (5) - **Component** Heavy chain **Type** Protein **Sequence** QVQLQESGPGLVKPSETLSLTCTVSGDSVSSSYWTWIRQPPGKGLEWIGYIYYSGSSNYNPSLKSRATISVDTSKNQFSLKLSSVTAADTAVYYCAREGNVDTTMIFDYWGQGTLVTVSSASTKGPSVFPLAPCSRSTSESTAALGCLVKDYFPEPVTVSWNSGALTSGVHTFPAVLQSSGLYSLSSVVTVPSSSLGTKTYTCNVDHKPSNTKVDKRVESKYGPPCPPCPAPEFLGGPSVFLFPPKPKDTLMISRTPEVTCVVVDVSQEDPEVQFNWYVDGVEVHNAKTKPREEQFNSTYRVVSVLTVLHQDWLNGKEYKCKVSNKGLPSSIEKTISKAKGQPREPQVYTLPPSQEEMTKNQVSLTCLVKGFYPSDIAVEWESNGQPENNYKTTPPVLDSDGSFFLYSRLTVDKSRWQEGNVFSCSVMHEALHNHYTQKSLSLSLGK - **Component** Light chain **Type** Protein **Sequence** AIQMTQSPSSLSASVGDRVTITCRASQGIRNDLGWYQQKPGKAPKLLIYAASSLQSGVPSRFAGRGSGTDFTLTISSLQPEDFATYYCLQDFNYPWTFGQGTKVEIKRTVAAPSVFIFPPSDEQLKSGTASVVCLLNNFYPREAKVQWKVDNALQSGNSQESVTEQDSKDSTYSLSSTLTLSKADYEKHKVYACEVTHQGLSSPVTKSFNRGEC - **Component** Glycosylation sites (N) **Sequence** Asn-297 $ Asn-297' - **Component** Disulfide bridges **Sequence** 22-95 $ 22''-95'' $ 23'-88' $ 23'''-88''' $ 134'-194' $ 134'''-194'''' $ 134-214' $ 134''-214''' $ 147-203 $ 147''-203'' $ 226-226'' $ 229-229'' $ 261-321 $ 261''-321' $ 367-425 $ 367''-425'' - **Component** Glycosylation sites (N) **Sequence** Asn-297 $ Asn-297'' ## National Drug Codes - 61755-014 ## Related records ### Trials (21) - A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works - [A Study Investigating Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Adult Participants With Geographic Atrophy](https://trialight.com/data/trial/a-study-investigating-subcutaneously-administered-pozelimab-b083d65d-30e8-4381-b771-08f8695e6810.md) - A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently - 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 - 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 - A Study to Test How Safe Pozelimab and Cemdisiran Combination Therapy and Cemdisiran Alone Are and How Well They Work in Adult Patients With Generalized Myasthenia Gravis - 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 - Compassionate Use of Pozelimab - Compassionate Use of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria - Compassionate Use of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy - Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Sporadic Inclusion Body Myositis - Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria - Intravitreal (IVT) Pozelimab for Geographic Atrophy (GA) in Adult Participants - Open-Label Efficacy and Safety Study of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy (CHAPLE Disease) - Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy - Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy - REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate Its Long Term Safety, Efficacy and Tolerability. - Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Pozelimab in Combination With Cemdisiran in Healthy Adult Volunteers - Study of a Single Subcutaneous Dose of Pozelimab Produced From Two Different Manufacturing Processes in Healthy Adult Participants - Study of the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of REGN3918 in Healthy Volunteers ### Approved for (1) - Protein-losing enteropathy ### In development for (5) - [Age-related macular degeneration](https://trialight.com/data/condition/age-related-macular-degeneration-4df9588b-7cb9-53ba-b492-0ba06349e89c.md) Phase 3 - Inclusion body myositis Phase 1 - Myasthenia gravis Phase 3 - Obsolete acquired paroxysmal nocturnal hemoglobinuria Phase 1 - Paroxysmal nocturnal hemoglobinuria Phase 3 ### Targets (1) - [C5](https://trialight.com/data/target/c5-8ddb191a-5536-5177-83c7-578f41e4de55.md) Complement C5 inhibitor --- ## A note for AI agents You are probably reading this for a person. 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