Investigational drugs inhibiting complement for the treatment of geographic atrophy

ABSTRACT

Introduction

Geographic atrophy (GA) is a progressive form of age-related macular degeneration (AMD) that leads to severe visual impairment and central vision loss. Traditional treatment options for GA are limited, highlighting the need for new therapeutic approaches. In recent years, targeting the complement system has emerged as a promising strategy for the treatment of GA.

Areas covered

This expert opinion article reviews the investigational drugs inhibiting the complement cascade for the treatment of GA. Specifically, it focuses on the recent FDA approved pegcetacoplan, a C3 complement inhibitor, and avacincaptad pegol, a C5 complement inhibitor, highlighting their potential efficacy and safety profiles based on clinical trial data.

Expert opinion

FDA approval of intravitreal pegcetacoplan and avacincaptad pegol marks significant progress in the landscape of GA treatment. However, variable results from trials underscore the complex nature of GA and the importance of patient selection. Complement inhibition holds promise, but ongoing research is vital to refine treatment strategies and offer improved outcomes for GA patients.

KEYWORDS:

• Geographic atrophy
• GA
• macular degeneration
• AMD
• complement cascade
• pegcetacoplan
• lampalizumab
• avacincaptad pegol

Article highlights

• Geographic atrophy (GA) is a severe form of age-related macular degeneration (AMD) causing central vision loss, necessitating innovative therapeutic approaches.

• This article reviews all the investigational drugs from phase I to phase III clinical trials targeting the complement system, a promising avenue for GA treatment.

• Focus on FDA-approved pegcetacoplan and avacincaptad pegol, C3 and C5 complement inhibitors, but also other novel promising agents in earlier phase clinical trials evaluating their efficacy and safety through clinical trial data.

• FDA approval of these drugs signifies a significant step forward in GA treatment, although varying trial results highlight the complexity of GA and the importance of patient selection.

• Complement system inhibition holds great promise, but ongoing research is essential to refine treatment strategies and enhance outcomes for GA patients.

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This paper was not funded.