Pozelimab
Also known as Veopoz, Pozelimab-bbfg
REGENERON PHARMACEUTICALS, INC. REGN · Paroxysmal Nocturnal Hemoglobinuria; Age-related Macular Degeneration (AMD); CD55-deficient Protein-losing Enteropathy; CHAPLE; Generalized Myasthenia Gravis
Key figures
Next catalyst
None scheduled
Only dates with a source are listed
Phase
Approved
Not recorded
Linked trials
7
From ClinicalTrials.gov
Latest FDA action
Approved
BLA761339,
Recalls, shortages, patents and EU status at a glance
Key information
- Sponsor
- REGENERON PHARMACEUTICALS, INC. REGN Source: Company record for REGENERON PHARMACEUTICALS, INC. (opens in a new tab)
- Indications
- Paroxysmal Nocturnal Hemoglobinuria; Age-related Macular Degeneration (AMD); CD55-deficient Protein-losing Enteropathy; CHAPLE; Generalized Myasthenia Gravis Source: Conditions from ClinicalTrials.gov (opens in a new tab)
- Application
- BLA 761339 · Priority review Source: Drugs@FDA via openFDA (opens in a new tab) · Action letter for BLA 761339 (opens in a new tab)
- Designations
- None recorded
- Next catalyst
- None scheduled
Development timeline
- Preclinical Completed
- Phase 1 Completed
- Phase 2 CompletedFirst trial start 27 Jan 20201 linked trial
- Phase 3 CompletedFirst trial start 27 Jan 20207 linked trials
- Filed Completed
- Approved Current stageFirst approval 18 Aug 2023
Overview
- Mechanism
- Not recorded
- Route
- Not recorded
- Modality
- Not recorded
- Specialties
- Not recorded
Overview text is written by HealthTrade editors.
Key dates
No catalysts yet. Editors add each date together with its source.
FDA designations
No FDA designations are on record for Pozelimab. Editors add each one with its source.
Filed application
No filed application is on record for Pozelimab. Approvals are listed under FDA actions.
FDA advisory committees
No FDA advisory committee meetings are on record for Pozelimab.
Clinical trials
| Trial | Title | Phase | Status | Enrollment | Primary completion | Results posted |
|---|---|---|---|---|---|---|
| NCT07154745ClinicalTrials.gov record for NCT07154745 (opens in a new tab) | 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 | Phase 3 | Recruiting | 35 (estimated) | 18 Jan 2030 (estimated) | No |
| NCT06541704ClinicalTrials.gov record for NCT06541704 (opens in a new tab)SIENNA | A Study Investigating Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Adult Participants With Geographic Atrophy | Phase 3 | Recruiting | 975 (estimated) | 31 Jul 2028 (estimated) | No |
| NCT05744921ClinicalTrials.gov record for NCT05744921 (opens in a new tab)ACCESS-EXT | 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 | Phase 3 | Recruiting | 202 (estimated) | 10 Oct 2028 (estimated) | No |
| NCT05131204ClinicalTrials.gov record for NCT05131204 (opens in a new tab)ACCESS 2 | Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria | Phase 3 | Terminated | 3 (actual) | 12 Jul 2023 (actual) | Yes First posted Results on ClinicalTrials.gov for NCT05131204 (opens in a new tab) |
Posted results on ClinicalTrials.gov for ACCESS 2 Serious adverse events (From first dose to week 29): Pozelimab and Cemdisiran 0 of 2; Anti-C5 Standard-of-care 0 of 1 Source: ClinicalTrials.gov results for NCT05131204 (opens in a new tab)· imported Posted results as ClinicalTrials.gov prints them: primary outcome analyses and serious adverse event totals only. Registry facts, not reviewed by HealthTrade and not investment advice; filing-derived results are listed separately. | ||||||
| NCT05133531ClinicalTrials.gov record for NCT05133531 (opens in a new tab)ACCESS-1 | 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 | Phase 3 | Active, not recruiting | 202 (actual) | 12 Oct 2026 (estimated) | No |
| NCT05070858ClinicalTrials.gov record for NCT05070858 (opens in a new tab)NIMBLE | 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 | Phase 3 | Active, not recruiting | 288 (actual) | 8 Jul 2025 (actual) | No |
Publications listing NIMBLE (1), newest first
Citations from PubMed, U.S. National Library of Medicine; abstracts are not stored. A publication lists this study’s NCT number and may be a review that cites many studies. | ||||||
| NCT04209634ClinicalTrials.gov record for NCT04209634 (opens in a new tab) | Open-Label Efficacy and Safety Study of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy (CHAPLE Disease) | Phase 2/3 | Completed | 10 (actual) | 9 Nov 2021 (actual) | Yes First posted Results on ClinicalTrials.gov for NCT04209634 (opens in a new tab) |
Posted results on ClinicalTrials.gov for NCT04209634 Serious adverse events (From signing of informed consent up to 164 weeks (end of study)): Pozelimab Injection 5 of 10 Source: ClinicalTrials.gov results for NCT04209634 (opens in a new tab)· imported Posted results as ClinicalTrials.gov prints them: primary outcome analyses and serious adverse event totals only. Registry facts, not reviewed by HealthTrade and not investment advice; filing-derived results are listed separately. Publications listing NCT04209634 (4), newest first
Citations from PubMed, U.S. National Library of Medicine; abstracts are not stored. A publication lists this study’s NCT number and may be a review that cites many studies. | ||||||
Source: ClinicalTrials.gov, U.S. National Library of Medicine. Posted results are shown as ClinicalTrials.gov prints them; results from company and journal sources are entered by editors and listed separately. Full result tables are on ClinicalTrials.gov.
Trial quality
About CT-VCALoading trial quality…
FDA actions
| Application | Action | Date | Outcome | Review | Source |
|---|---|---|---|---|---|
| BLA761339Action letter for BLA761339 (opens in a new tab) | Original 1 Type 1 - New Molecular Entity | Approved | priority | Drugs@FDA via openFDA (opens in a new tab) |
Data provided by the U.S. Food and Drug Administration.
FDA recalls load here.
FDA drug shortages load here.
Orange Book patents load here.
FAERS adverse event counts load here.
Papers and preprints load here.
Community discussion loads here.
EU status (EMA)
EMA's medicines data lists no human medicine with the same active substance as Pozelimab.
Source: European Medicines Agency (EMA), medicines data download.
Related news
No recent headlines mention this drug.
Sources
- Drugs@FDA 761339 (opens in a new tab) Licence: public-domain. Official registry record this listing came from
- ClinicalTrials.gov NCT07154745 (opens in a new tab) Licence: public-domain. Fetched
- ClinicalTrials.gov NCT06541704 (opens in a new tab) Licence: public-domain. Fetched
- ClinicalTrials.gov NCT05744921 (opens in a new tab) Licence: public-domain. Fetched
- ClinicalTrials.gov NCT05131204 (opens in a new tab) Licence: public-domain. Fetched
- ClinicalTrials.gov NCT05133531 (opens in a new tab) Licence: public-domain. Fetched
- ClinicalTrials.gov NCT05070858 (opens in a new tab) Licence: public-domain. Fetched
- ClinicalTrials.gov NCT04209634 (opens in a new tab) Licence: public-domain. Fetched
- Drugs@FDA via openFDA BLA761339 (opens in a new tab) Licence: cc0. Fetched
Record updated .
Dates come from company and FDA disclosures and can change. Nothing on this page is investment advice.