Database for
Therapeutic Antibodies
Therapeutic Antibodies
| Entry ID | 122 |
| INN | Pozelimab |
| Status | Approved |
| Drug code(s) | REGN3918 |
| Brand name | VEOPOZ |
| mAb sequence source | mAb human |
| General Molecular Category | Naked monospecific |
| Format, general category | Full length Ab |
| Format details | None |
| Isotype (Fc) | IgG4 |
| Light chain isotype | kappa |
| Linker | None |
| Ave. DAR | None |
| Conjugated/fused moiety | None |
| Discovery method/technology | Transgenic mouse |
| Target(s) | Complement C5 |
| Indications of clinical studies | Generalized Myasthenia Gravis, CD55-deficient Protein-losing Enteropathy, Paroxysmal nocturnal hemoglobinuria, Phase 1 in health volunteers, intended for ocular inflammatory and retinal degenerative diseases |
| Primary therapeutic area | Cardiovascular / hemostasis disorders |
| Most advanced stage of development (global) | Approved US |
| Status | Active |
| Start of clinical phase (IND filing or first Phase 1) | May 15, 2017 |
| Start of Phase 2 | May 16, 2019 |
| Start of Phase 3 | December 03, 2019 |
| Date BLA/NDA submitted to FDA | December 20, 2022 |
| Year of first approval (global) | 2023 |
| Date of first US approval | August 18, 2023 |
| INN, US product name | Pozelimab, pozelimab-bbfg |
| US or EU approved indications | Treatment of adult and pediatric patients 1 year of age and older with CD55-deficient protein-losing enteropathy (PLE), also known as CHAPLE disease. ( |
| Company | Regeneron Pharmaceuticals |
| Licensee/Partner | None |
| Comments about company or candidate | Feb 21, 2023: Regeneron Pharmaceuticals, Inc. announced the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) for pozelimab as a treatment for adults and children as young as 1 year of age with CHAPLE disease (also known as CD55 deficiency with Hyperactivation of complement, Angiopathic thrombosis and Protein Losing Enteropathy or CD55-deficient protein-losing enteropathy). PDUFA date is August 20, 2023. Aug 2022 corporate update: BLA in CD55-deficient protein-losing enteropathy anticipated in H2 2022. Pozelimab was also granted Fast Track designation in September 2022 The FDA designated pozelimab for treatment of CHAPLE as a drug for a “rare pediatric disease” in April 2020; FDA has granted pozelimab Orphan Drug designations for the treatment of CD55-deficient protein-losing enteropathy and paroxysmal nocturnal hemoglobinuria. NCT04491838 Phase 1 of subcutaneous administration. NCT04209634 Phase 2/3 in CD55-deficient Protein-losing Enteropathy not yet recruiting as of Jan 1 2020. NCT04162470 Phase 3 in Paroxysmal Nocturnal Hemoglobinuria enrolling by invitation stating Dec 3 2019. NCT03946748 Phase 2 study in PNH recruiting as of May 16, 2019. Phase 1 NCT03115996 study still recruting as of July 2018. The Company expects to report full data from its Phase 1 study in paroxysmal nocturnal hemoglobinuria (PNH) in the second half of 2018, and plans to initiate a Phase 2 study in PNH in early 2019. |
| Full address of company | Tarrytown, New York, United States North America United States of America https://www.regeneron.com/ |
None
| Anticipated events | None |
| Factor(s) contributing to discontinuation | None |