Overview
CD55-deficient protein-losing enteropathy (PLE), or CHAPLE disease, is an ultra-rare hereditary disease, with fewer than 100 patients diagnosed worldwide or fewer than 10 patients in the US. The pathophysiology of this disease is mainly attributed to the deficiency of the CD55 protein, which is the main regulator of the complement cascade.. Under normal circumstances, CD55 inhibits the activity of C3 and C5 convertases, thus preventing the cleavage of C3 and C5 respectively into immunoreactive peptides C3a and C5a. The loss of CD55 can therefore induce complement hyperactivation, causing the unwanted formation of membrane-attacking complex and resulting in paroxysmal nocturnal hemoglobinuria and complement-mediated autoimmune hemolysis that are often observed in CHAPLE disease. Pozelimab is a human, monoclonal immunoglobulin G4 antibody against the terminal complement protein C5. In August 18, 2023, pozelimab was approved by the FDA for the treatment of CHAPLE disease. It is currently the only treatment explicitly indicated for CHAPLE disease.
Indication
Pozelimab is indicated for the treatment of adult and pediatric patients 1 year of age and older with CD55-deficient protein-losing enteropathy (PLE), also known as CHAPLE disease.
Associated Conditions
- CHAPLE disease
Research Report
Pozelimab (Veopoz): A Comprehensive Monograph on a First-in-Class C5 Inhibitor for Complement-Mediated Disease
Section 1: Profile of a Novel Complement C5 Inhibitor
Pozelimab represents a significant advancement in the targeted modulation of the human immune system. As a high-precision biologic therapeutic, it is designed to intervene at a critical juncture of the complement cascade, a powerful yet potentially destructive component of innate immunity. This section establishes the fundamental identity of pozelimab, detailing its nomenclature, molecular architecture, and pharmacological classification, which together form the basis for its clinical application.
1.1 Identification and Nomenclature
To ensure clarity and precision across clinical, regulatory, and research domains, pozelimab is identified by a comprehensive set of names and codes.
- Nonproprietary Names: The International Nonproprietary Name (INN) assigned to the molecule is Pozelimab.[1] In the United States, the approved nonproprietary name, which includes a four-letter suffix to distinguish it from other biological products, is pozelimab-bbfg.[2]
- Brand Name: The commercial product is marketed under the trade name Veopoz.[2]
- Development and Regulatory Codes: During its development by Regeneron Pharmaceuticals, the antibody was designated REGN3918.[4] Its United States Adopted Name (USAN) council code is FG-06.[1]
- Database and Chemical Identifiers: Pozelimab is cataloged in major scientific databases under the following unique identifiers:
- DrugBank Accession Number: DB15218 [2]
- CAS (Chemical Abstracts Service) Registry Number: 2096328-94-6 [2]
- UNII (Unique Ingredient Identifier): 0JJ21K6L2I [1]
The following table consolidates these key identifiers and properties, providing a definitive reference for the molecule.
Clinical Trials
Title | Posted | Study ID | Phase | Status | Sponsor |
|---|---|---|---|---|---|
2025/09/04 | Not Applicable | Not yet recruiting | |||
2025/08/26 | Not Applicable | Not yet recruiting | |||
2024/08/07 | Phase 3 | Recruiting | |||
2023/09/08 | N/A | AVAILABLE | |||
2023/08/22 | N/A | AVAILABLE | |||
2023/02/27 | Phase 3 | Recruiting | |||
2022/02/09 | N/A | AVAILABLE | |||
2021/11/24 | Phase 3 | Recruiting | |||
2021/11/23 | Phase 3 | Terminated | |||
2021/10/07 | Phase 3 | Active, not recruiting |
FDA Drug Approvals
Approved Product | Manufacturer | NDC Code | Route | Strength | Effective Date |
|---|---|---|---|---|---|
| No FDA approvals found for this drug. | |||||
EMA Drug Approvals
Approved Product | Authorization Holder | Status | Issued Date |
|---|---|---|---|
| No EMA approvals found for this drug. | |||
HSA Drug Approvals
Approved Product | Manufacturer | Approval Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| No HSA approvals found for this drug. | |||||
NMPA Drug Approvals
Approved Product | Company | Approval Number | Drug Type | Dosage Form | Approval Date |
|---|---|---|---|---|---|
| No NMPA approvals found for this drug. | |||||
PPB Drug Approvals
Approved Product | Registration No. | Company | Licence No. | Strength | Registration Date |
|---|---|---|---|---|---|
| No PPB approvals found for this drug. | |||||
TGA Drug Approvals
Approved Product | ARTG ID | Sponsor | Registration Type | Status | Registration Date |
|---|---|---|---|---|---|
| No TGA approvals found for this drug. | |||||
Health Canada Drug Approvals
Approved Product | Company | DIN | Dosage Form | Strength | Market Date |
|---|---|---|---|---|---|
| No Health Canada approvals found for this drug. | |||||
CIMA AEMPS Drug Approvals
Approved Product | Company | Registration Number | Pharmaceutical Form | Prescription Type | Status |
|---|---|---|---|---|---|
| No CIMA AEMPS (Spain) approvals found for this drug. | |||||
Philippines FDA Drug Approvals
Approved Product | Company | License Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| No Philippines FDA approvals found for this drug. | |||||
Saudi SFDA Drug Approvals
Approved Product | Company | License Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| No Saudi SFDA approvals found for this drug. | |||||
Malaysia NPRA Drug Approvals
Approved Product | Company | Registration Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| No Malaysia NPRA approvals found for this drug. | |||||
UK EMC Drug Information
Medicine Name | MA Holder | MA Number | Pharmaceutical Form | Active Ingredient | Authorization Date |
|---|---|---|---|---|---|
| No UK EMC drug information found for this drug. | |||||
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