Annexon has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for tanruprubart for the treatment of Guillain-Barré syndrome (GBS).

Annexon is the developer of vonaprument, which is in phase 3 development for the treatment of dry age-related macular degeneration (AMD) with geographic atrophy (GA). Vonaprument is a first-in-kind, non-pegylated antigen-binding fragment (Fab) designed to block C1q locally in the eye with an intravitreal formulation. 

The new submission marks Annexon’s first regulatory filing and represents a milestone for patients with GBS, a rare but life-threatening acute neuroinflammatory disease that can rapidly lead to severe weakness, paralysis, and respiratory failure. GBS affects at least 150,000 people worldwide each year, and there are currently no FDA-approved disease-modifying therapies.

“Annexon’s first regulatory submission marks a defining milestone for patients and for the company and represents the first of several important registrational catalysts anticipated from our lead programs in 2026,” said Douglas Love, president and chief executive officer of Annexon. “In the landmark phase 3 study, tanruprubart was shown to rapidly stop neuroinflammation, enabling GBS patients to recover faster and more completely from this sudden, life-threatening disease that has no approved disease-modifying therapies.”

Tanruprubart is an investigational, first-in-class monoclonal antibody designed to selectively block classical complement-driven inflammation, a key mechanism underlying nerve damage in GBS. Unlike current standard-of-care treatments such as intravenous immunoglobulin (IVIg) or plasma exchange—which require intensive, multi-day administration and provide incomplete benefit—tanruprubart has been observed to act almost immediately following a single infusion by halting early nerve injury.

A Biologics License Application (BLA) submission to the FDA, incorporating data from the FORWARD study, is planned for 2026.