Methods of treating ttp with immunoglobulin single variable domains and uses thereof

Abstract

The present invention is based on the finding that administration of polypeptides comprising at least one Immunoglobulin single variable domains against vWF to human TTP patients provides a significant decrease in the time to response. The invention provides a polypeptide comprising at least one immunoglobulin single variable domain (ISVD) against von Willebrand Factor (vWF) for use in treating a vWF-related disease in a human in need thereof. The invention further relates to dosage unit forms, kits and medical uses for treating TTP.

Description

RELATED APPLICATIONS[0001]This application is a continuation of U.S. application Ser. No. 15 / 319,467, filed Dec. 16, 2016, which is a national stage filing under 35 U.S.C. § 371 of International Application No. PCT / EP2015 / 063493, filed Jun. 16, 2015, which was published under PCT Article 21(2) in English, and claims the benefit under 35 U.S.C. § 119(e) of U.S. provisional application Ser. No. 62 / 030,817, filed Jul. 30, 2014, the entire contents of each of which is incorporated by reference herein in its entirety.1. FIELD OF THE INVENTION[0002]The present invention is based on the finding that administration of polypeptides comprising at least one Immunoglobulin single variable domain against vWF to human TTP patients provides a significant decrease in the time to response and less complications. The invention provides a polypeptide comprising at least one immunoglobulin single variable domain (ISVD) against von Willebrand Factor (vWF) for use in treating a vWF-related disease in a h...

AI Technical Summary

Benefits of technology

[0019]Moreover, the present clinical study with TTP patients also demonstrates that the polypeptides of the present invention (e.g. ALX 0081) are well tolerated agents and, in particular, that the potential for the risk of bleeding appears to be present but low and manageable. The currently available data demonstrate, therefore, that the reduction in PE and transfusion and their associated complications are achieved without significant adverse effects from the use of the polypeptides of the invention itself. This represents a clear safety benefit for the use of the polypeptides of the invention in the treatment of patients with TTP.

[0172]In an embodiment, the present invention relates to a method of reducing the likelihood of a human contracting ischaemic organ damage by a vWF-related disease, which comprises administering to the human at a predefined dose a polypeptide comprising at least one immunoglobulin single variable domain (ISVD) against von Willebrand Factor (vWF), wherein each administration of the antibody delivers to the human from 0.1 mg per kg to 25 mg per kg of the human's body weight, so as to thereby reduce the likelihood of the human contracting ischaemic organ damage.

Problems solved by technology

However, this treatment requires multiple exchanges and transfusions over many days, during which time there is no direct pharmacological targeting of the active process of ULvWF-mediated platelet aggregation.

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