Database for
Therapeutic Antibodies
Therapeutic Antibodies
| Entry ID | 8 |
| INN | Emicizumab |
| Status | Approved |
| Drug code(s) | ACE910, RO5534262, RG6013 |
| Brand name | Hemlibra |
| mAb sequence source | mAb humanized |
| General Molecular Category | Bispecific |
| 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 | None |
| Target(s) | Factor IXa, Factor X |
| Indications of clinical studies | Hemophilia A |
| Primary therapeutic area | Cardiovascular / hemostasis disorders |
| Most advanced stage of development (global) | Approved EU, US, Japan, Australia |
| Status | Active |
| Start of clinical phase (IND filing or first Phase 1) | August 15, 2012 |
| Start of Phase 2 | |
| Start of Phase 3 | November 18, 2015 |
| Date BLA/NDA submitted to FDA | June 23, 2017 |
| Year of first approval (global) | 2017 |
| Date of first US approval | November 16, 2017 |
| INN, US product name | Emicizumab, emicizumab-kxwh |
| US or EU approved indications | Hemophilia A (routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with hemophilia A with factor VIII inhibitors). Approved in March 2019 in EU for treatment of patients with severe hemophilia without factor VIII inhibitors |
| Company | Chugai Pharmaceuticals |
| Licensee/Partner | None |
| Comments about company or candidate | Approved on November 16, 2017 in US; Approved in EU Feb 2018; approved in Japan Priority review in US and accelerated assessment in EU; PDUFA date Feb 23 2018. Chugai's submission of marketing application in Japan announced July 21, 2017. NCT02622321 Phase 3 study in patients with congenital hemophilia A. Breakthrough Therapy Designation for the prophylactic treatment of people who are 12 years or older with hemophilia A with factor VIII inhibitors. June 2015: Data from Phase I extension study presented at major medical conference on bleeding disorders showed that reduction in bleeding rates was maintained after a follow-up of 5.6 to 18.5 months; ACE910 is the first FVIIIa-mimetic bispecific antibody to have shown potential benefit in people with severe haemophilia A both with or without inhibitors to factor VIII. Orphan designations in Japan, EU and US; listed in Roche pipeline as Phase 2 as of April 2014. First-in-human study published in Blood. 2016 Mar 31;127(13):1633-41; this trial was registered at www.clinicaltrials.jp as #JapicCTI-121934, duration of study listed as 2012-8 ～ 2015-8. |
| Full address of company | 1-1 Nihonbashi-Muromachi 2-Chome Chuo-ku, Tokyo 103-8324 JAPAN Asia Japan https://www.chugai-pharm.co.jp/english/rule/contact/index.html |
cLC-hetero-H-chain IgG. The CDRs of the anti-FIXa heavy chain (Q chain), the anti-FX heavy chain (J chain), and the light chains were derived from rats immunized with human FIXa, mice immunized with human FX, and both, respectively. Emicizumab molecule contains two different heavy chains and two identical light chains. S228P; K196Q; F296Y; E356K; K439E; L445P; G446 > del; K447 > del
| Anticipated events | None |
| Factor(s) contributing to discontinuation | None |