Database for
Therapeutic Antibodies
Therapeutic Antibodies
| Entry ID | 92 |
| INN | Burosumab |
| Status | Approved |
| Drug code(s) | KRN23 |
| Brand name | Crysvita |
| 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 | kappa |
| Linker | None |
| Ave. DAR | None |
| Conjugated/fused moiety | None |
| Discovery method/technology | Transgenic mouse (KM mouse) |
| Target(s) | FGF23 |
| Indications of clinical studies | X-linked Hypophosphatemia |
| Primary therapeutic area | Skeletal disorders |
| Most advanced stage of development (global) | Approved EU, US, Japan, Australia |
| Status | Active |
| Start of clinical phase (IND filing or first Phase 1) | October 15, 2008 |
| Start of Phase 2 | June 15, 2014 |
| Start of Phase 3 | December 01, 2015 |
| Date BLA/NDA submitted to FDA | August 17, 2017 |
| Year of first approval (global) | 2018 |
| Date of first US approval | April 17, 2018 |
| INN, US product name | Burosumab, burosomab-twza |
| US or EU approved indications | Treatment of patients age two and older with tumor-induced osteomalacia, X-linked Hypophosphatemia with radiographic evidence of bone disease in children aged 1 and older, and in adolescents with growing skeletons |
| Company | Kyowa Hakko Kirin |
| Licensee/Partner | Ultragenyx Pharmaceutical |
| Comments about company or candidate | PDUFA date April 17, 2018. 2018: Ultragenyx awarded rare pediatric disease designation prioity review voucher for Crysvita (burosumab-twza) to treat X-linked hypophosphatemia (XLH). Sold to an undisclosed party for $80.6 million. June 23, 2017: Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that it has reached agreement with the FDA at a Pre-Biologics License Application (pre-BLA) meeting on the clinical package to support the burosumab (KRN23) BLA filing for X-linked hypophosphatemia (XLH). At the meeting, the FDA agreed that the BLA can be submitted based on available clinical data and confirmed that both pediatric and adult indications would be included in the review. Based on the agreement, the submission of the burosumab BLA is planned for the second half of 2017. Breakthrough therapy designation for pediatric XLH; US orphan drug designation. NCT02526160 Phase 3 study and NCT02537431 Phase 3 study recruiting as of Dec 2015. Collaborator is Ultragenyx Pharmaceutical as of Sept 2013. Ultragenyx and Kyowa Hakko Kirin initiated a pediatric X-linked hypophosphatemia (XLH) program using KRN23 in 2014. Ultragenyx Pharmaceutical announced that the European Commission has granted orphan medicinal product designation for recombinant human monoclonal IgG1 antibody for fibroblast growth factor 23 (KRN23 or UX023) for the treatment of X-linked hypophosphatemia (XLH). |
| Full address of company | Tokyo, Japan Asia Japan https://www.kyowakirin.com/index.html |
None
| Anticipated events | None |
| Factor(s) contributing to discontinuation | None |