Database for
Therapeutic Antibodies
Therapeutic Antibodies
| Entry ID | 88 |
| INN | Pabinafusp alfa |
| Status | Approved |
| Drug code(s) | JR-141 |
| Brand name | IZCARGO |
| mAb sequence source | mAb humanized |
| General Molecular Category | Immunoconjugate |
| Format, general category | Full length Ab fusion |
| Format details | None |
| Isotype (Fc) | IgG1 |
| Light chain isotype | kappa |
| Linker | None |
| Ave. DAR | None |
| Conjugated/fused moiety | IDS |
| Discovery method/technology | None |
| Target(s) | TfR |
| Indications of clinical studies | Hunter syndrome (mucopolysaccharidosis II) |
| Primary therapeutic area | Metabolic disorders |
| Most advanced stage of development (global) | Approved Japan |
| Status | Active |
| Start of clinical phase (IND filing or first Phase 1) | March 30, 2017 |
| Start of Phase 2 | July 26, 2018 |
| Start of Phase 3 | |
| Date BLA/NDA submitted to FDA | |
| Year of first approval (global) | 2021 |
| Date of first US approval | |
| INN, US product name | Pabinafusp alfa |
| US or EU approved indications | None |
| Company | JCR Pharmaceuticals Co. Ltd. |
| Licensee/Partner | Takeda |
| Comments about company or candidate | Sep 2021: Under the terms of the exclusive collaboration and license agreement, Takeda will exclusively commercialize JR-141 outside of the United States, including Canada, Europe, and other regions (excluding Japan and certain other Asia-Pacific countries). March 23, 2021 JCR Pharmaceuticals Co., Ltd. (TSE 4552; "JCR") today announced that the Ministry of Health, Labour and Welfare (MHLW) in Japan has approved IZCARGO® (pabinafusp alfa 10 mL, intravenous drip infusion) for the treatment of mucopolysaccharidosis type II (MPS II, or Hunter syndrome). IZCARGO® (formerly known as JR-141) is a recombinant iduronate-2-sulfatase enzyme replacement therapy (ERT) that relies on J-Brain Cargo®, a proprietary technology developed by JCR, to deliver therapeutics across the blood-brain barrier (BBB). It is the first-ever approved ERT that penetrates the BBB via intravenous administration, a potentially life-changing benefit for individuals with lysosomal storage disorders (LSDs) such as MPS II. Approved in Japan under SAKIGAKE designation is a key milestone in JCR Pharmaceuticals’ global expansion. Fast Track designation from the US FDA, orphan designation from the European Medicines Agency NCT04573023 Phase 3 due to start in Aug 2021. Results of Phase 2 study in Brazil: https://www.sciencedirect.com/science/article/pii/S1525001621001489 |
| Full address of company | Ashiya, Hyogo, Japan Asia Japan https://www.jcrpharm.co.jp/en/site/en/ |
Pabinafusp alfa (JR-141) consists of human iduronate-2-sulfatase (IDS) fused to the C terminus of the heavy chain of an anti-human transferrin receptor (hTfR) antibody
| Anticipated events | None |
| Factor(s) contributing to discontinuation | None |