Caplacizumab – anti-vWF NANOBODY® to treat acquired Thrombotic Thrombocytopenic Purpura (aTTP)

On 3 September 2018, caplacizumab was approved in Europe for adults with acquired thrombotic thrombocytopenic purpura (aTTP) - press release. Caplacizumab has been launched in Germany in October 2018.

Moreover, on 6 February 2019, the FDA approved (caplacizumab, the first NANOBODY®-based medicine, for adults with aTTP. It is the first medicine approved in the U.S. specifically for the treatment of aTTP, a rare blood-clotting disorder - press release.

Caplacizumab was developed by Ablynx, which was acquired by Sanofi in 2018. Caplacizumab is part of the company’s rare blood disorders franchise within Sanofi Genzyme, the specialty care global business unit of Sanofi.

acquired Thrombotic Thrombocytopenic Purpura (aTTP)

Acquired TTP is an ultra-rare, acute, life-threatening blood clotting disorder that leads to extensive micro-clot formation in the small blood vessels throughout the body and tissue ischaemia and damage to vital organs, including the heart, brain and kidneys. Mortality is high at 10-20%[2], with vast majority within 2 weeks post diagnosis, and about 36% of patients suffer from recurrences [1] after initial treatment with the current standard-of-care, which consists of plasma exchange (PE) plus immune-suppressive treatment. In addition, the organ damage caused by a TTP episode may result in poor longer term outcomes.

There remained a high unmet medical need to immediately inhibit the formation of microvascular thrombi, thereby reducing the risk of further organ damage. Maintenance of this platelet-protective effect is required until the underlying auto-immune activity has been resolved. Caplacizumab has been developed to address this unmet need.

[1] George et al, 2008
[2] Scully et al, Br J Haematology 2012

Related publications, presentations, abstracts and posters

Presentation: "Results of the Randomized, Double-Blind, Placebo-Controlled, Phase 3 Hercules Study of Caplacizumab in Patients with Acquired Thrombotic Thrombocytopenic Purpura", presented by Professor Marie Scully in the late-breaking abstracts session at the 59th Annual Meeting of the American Society of Hematology (ASH) in Atlanta, GA, USA

Publication: Peyvandi, F., Scully, M., Kremer Hovinga, J. A., Knöbl, P., Cataland, S., De Beuf, K., Callewaert, F., De Winter, H. and Zeldin, R. K., Caplacizumab reduces the frequency of major thromboembolic events, exacerbations, and death in patients with acquired thrombotic thrombocytopenic purpura. J Thromb Haemost. Accepted Author Manuscript. doi:10.1111/jth.13716

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