Dosing regimens for oral complement factor D inhibitors

Abstract

Compounds and pharmaceutically acceptable salts and prodrugs thereof are disclosed, which are inhibitors of the complement system. Also provided are oral dosage forms comprising such compounds, salts, or prodrugs. Also disclosed are methods of using the compounds, salts and prodrugs and oral dosage forms thereof to treat or prevent diseases or conditions characterized by aberrant complement system activity (e.g., paroxysmal sleep hemoglobinuria).

Description

[0001] related application [0002] This application claims U.S. Provisional Patent Application No. 62 / 881,225, filed July 31, 2019; U.S. Provisional Patent Application No. 62 / 926,175, filed October 25, 2019; and U.S. Provisional Patent Application No. 62 / 926,175, filed May 5, 2020 Priority interest under No. 63 / 020,239. Background technique [0003] The complement system is a branch of an organism's immune system that enhances the ability of antibodies and phagocytes to destroy and remove foreign particles (eg, pathogens) from an organism. The complement system comprises a collection of plasma proteins that work together to attack the extracellular forms of pathogens and induce a cascade of inflammatory responses that help fight infection. Complement activation can occur through several pathways. For example, complement activation can occur spontaneously in response to certain pathogens, or through the binding of antibodies to the pathogen. When complement proteins are act...

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Problems solved by technology

The complement proteins then create holes, or pores, in the invading organism, leading to its destruction

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