Complement in human disease: approved and up-and-coming therapeutics

Area of Science:

• Immunology
• Pharmacology

Background:

• The complement system is crucial for immune defense and tissue homeostasis.
• Dysregulated complement activity contributes to inflammatory and autoimmune diseases.
• Targeting the complement system for therapeutic benefit is a long-standing goal, but drug approval rates have been modest.

Purpose of the Study:

• To review the progress and challenges in developing complement therapeutics.
• To highlight novel targets and emerging technologies in complement drug development.
• To discuss the potential expansion of complement-targeted therapies to common diseases.

Main Methods:

• Review of scientific literature on complement system biology and therapeutics.
• Analysis of clinical trial data and drug approval trends.
• Discussion of advancements in drug discovery and development technologies.

Main Results:

• The anti-C5 antibody eculizumab has demonstrated success in treating paroxysmal nocturnal haemoglobinuria and atypical haemolytic syndrome.
• Increased understanding of complement pathways has identified new therapeutic targets.
• A surge in interest and development of novel complement therapeutics is evident.

Conclusions:

• Advances in complement biology and drug development technologies are driving innovation in complement therapeutics.
• While current approved drugs primarily target rare diseases, the pipeline suggests future opportunities for common inflammatory conditions.
• Targeting the complement system remains a promising strategy for managing a range of diseases.