Database for
Therapeutic Antibodies
Therapeutic Antibodies
| Entry ID | 15 |
| INN | Caplacizumab |
| Status | Approved |
| Drug code(s) | ALX-0081, ALX-0681 |
| Brand name | Cablivi |
| mAb sequence source | mAb humanized |
| General Molecular Category | Naked monospecific |
| Format, general category | Fragment |
| Format details | sdAb, VHH-VHH |
| Isotype (Fc) | None |
| Light chain isotype | None |
| Linker | None |
| Ave. DAR | None |
| Conjugated/fused moiety | None |
| Discovery method/technology | Camelid-derived; Phage display library |
| Target(s) | von Willebrand factor |
| Indications of clinical studies | Acquired Thrombotic Thrombocytopenic Purpura, Unstable Angina; Non-ST Segment Elevation Myocardial Infarction (NSTEMI); Stable Angina (Associated With High Risk PCI), Thrombotic Thrombocytopenic Purpura |
| Primary therapeutic area | Cardiovascular / hemostasis disorders |
| Most advanced stage of development (global) | Approved EU, US, Japan |
| Status | Active |
| Start of clinical phase (IND filing or first Phase 1) | February 15, 2007 |
| Start of Phase 2 | September 01, 2009 |
| Start of Phase 3 | October 01, 2015 |
| Date BLA/NDA submitted to FDA | June 06, 2018 |
| Year of first approval (global) | 2018 |
| Date of first US approval | February 06, 2019 |
| INN, US product name | Caplacizumab, caplacizumab-yhdp |
| US or EU approved indications | Acquired thrombotic thrombocytopenic purpura |
| Company | Sanofi |
| Licensee/Partner | None |
| Comments about company or candidate | Feb 6 2019: The U.S. Food and Drug Administration today approved Cablivi (caplacizumab-yhdp) injection, the first therapy specifically indicated, in combination with plasma exchange and immunosuppressive therapy, for the treatment of adult patients with acquired thrombotic thrombocytopenic purpura. Sep 3 2018. Sanofi said the European Commission approved Cablivi caplacizumab to treat rare blood clotting disorder acquired thrombotic thrombocytopenic purpura (aTTP). The company also revealed Monday that FDA accepted and granted Priority Review to its BLA for the drug; the PDUFA date is Feb. 6, 2019. Sanofi expects to file BLA in Q3 2018. Jan 2018: Ablynx acquired by Sanofi. July 2017: Ablynx's caplacizumab as a treatment for patients with acquired thrombotic thrombocytopenic purpura has been granted fast-track status by the FDA. MAA for acquired thrombotic trombocytopenic purpura submitted in Feb 2017. Company expects to submit BLA in 2018. NCT02553317 Phase 3 recruiting as of Oct 2015. The ability of caplacizumab to rapidly inhibit the formation of small blood clots, resulting in the more rapid restoration of normal platelet counts and an important reduction in exacerbations, was well demonstrated in the Phase II TITAN study. Based on the clinical effect seen in this TITAN study, we are planning to submit caplacizumab for conditional approval to the European Medicines Agency (EMA) in 2017. We now look forward to enrolling 92 patients into our Phase III “HERCULES” study which we plan to have completed by the end of 2017 to support a BLA filing in the United States in 2018. Orphan designation in US and EU. |
| Full address of company | Paris, France Europe France https://www.sanofi.us/en/contact-us |
VH-linker-VH fragment of Camelidae antibody; bivalent, no half-life extension
| Anticipated events | None |
| Factor(s) contributing to discontinuation | None |