Database for
Therapeutic Antibodies
Therapeutic Antibodies
| Entry ID | 1731 |
| INN | Marstacimab |
| Status | Approved |
| Drug code(s) | PF-06741086 |
| Brand name | Hympavzi* |
| mAb sequence source | mAb human |
| General Molecular Category | Naked monospecific |
| Format, general category | Full length Ab |
| Format details | None |
| Isotype (Fc) | IgG1 |
| Light chain isotype | lambda |
| Linker | None |
| Ave. DAR | None |
| Conjugated/fused moiety | None |
| Discovery method/technology | None |
| Target(s) | Tissue factor pathway inhibitor |
| Indications of clinical studies | Severe hemophilia, Phase 1 in healthy subjects |
| 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) | September 15, 2015 |
| Start of Phase 2 | March 08, 2017 |
| Start of Phase 3 | October 30, 2019 |
| Date BLA/NDA submitted to FDA | October 11, 2023 |
| Year of first approval (global) | 2024 |
| Date of first US approval | October 11, 2024 |
| INN, US product name | Marstacimab, marstacimab-hncq |
| US or EU approved indications | Routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and pediatric patients 12 years of age and older with hemophilia A (congenital factor VIII deficiency) without factor VIII (FVIII) inhibitors, or hemophilia B (congenital factor IX deficiency) without factor IX (FIX) inhibitors |
| Company | Pfizer |
| Licensee/Partner | None |
| Comments about company or candidate | Oct 11, 2024: Pfizer Inc. announced that the U.S. Food and Drug Administration (FDA) has approved HYMPAVZI™ (marstacimab-hncq) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and pediatric patients 12 years of age and older with hemophilia A (congenital factor VIII deficiency) without factor VIII (FVIII) inhibitors, or hemophilia B (congenital factor IX deficiency) without factor IX (FIX) inhibitors. Sep 20, 2024: Hympavzi* (marstacimab), received a positive opinion for the treatment of bleeding episodes in patients aged 12 years and older with severe haemophilia A or B, two types of a rare inherited bleeding disorder. The FDA has set a PDUFA action date in the fourth quarter of 2024, and a decision from the EC is anticipated by the first quarter of 2025. Mid-Dec 2023: Pfizer announces BLA submitted to FDA. Marstacimab appears on EMA list of applications under evaluation by the CHMP (Data extracted 30 October 2023) May 2023: Pfizer Inc. announced that the pivotal Phase 3 BASIS clinical trial (NCT03938792) evaluating marstacimab has met its primary endpoints. Analyses of the full Phase 3 dataset are ongoing, and results will be presented at an upcoming scientific conference. Pfizer will discuss these data with regulatory authorities, with the goal of initiating regulatory filings in the coming months. NCT05145127 is a Phase 3 with primary completion date in July 2030. NCT04878731 is a Phase 1 of subcutaneous formulation. NCT03938792 (EudraCT2018-003660-31) Phase 3 study in severe hemophilia A or B started in Oct 2019; primary completion date is in Sep 2024. NCT03363321 multicenter, open-label Phase II study to evaluate the long-term safety, tolerability and efficacy of subcutaneous or intravenous PF-06741086 in subjects with severe hemophilia started in May 2018. NCT02531815 Phase 1 study in health subjects completed in July 2016. US and EU orphan designations. Fast track designation in US. |
| Full address of company | 66 Hudson Boulevard East, New York, NY 10001-2192 USA North America United States of America https://www.pfizer.com |
Tissue factor pathway inhibitor (or TFPI) is a single-chain polypeptide which can reversibly inhibit Factor Xa (Xa). Fc mutations L234A, L235A, G237A (impair Fc effector functions)
| Anticipated events | Regulatory review EU |
| Factor(s) contributing to discontinuation | None |