Tanruprubart Succeeds in Late-Stage Guillain-Barré Trial
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- Tanruprubart demonstrated efficacy in a 241-person late-stage trial in Bangladesh and the Philippines, cutting mechanical ventilation time by 28 days, ICU stays by 7 days, and enabling independent walking 31 days earlier versus placebo, with a higher proportion reporting they felt "very much improved" after one week.
- Annexon Biosciences submitted tanruprubart to the European Medicines Agency and is preparing a US Food and Drug Administration submission in the coming months, with EU approval hoped for in early next year.
- The monoclonal antibody works by blocking the C1q protein to switch off the classical complement pathway, and no serious side effects or increased infection risk emerged despite suppressing part of the immune system — likely because it involves only a single infusion.
- Guillain-Barré syndrome causes the immune system to attack nerves after infections like food poisoning or influenza, leading to weakness, paralysis, or death, and affects roughly 8,000 people annually in the US with higher prevalence in low-income countries.
- Current GBS treatments — plasma exchange and intravenous immunoglobulin — are non-targeted and have seen no new options for 25 to 30 years, according to Stefan Blum at Princess Alexandra Hospital in Brisbane, who called them "useful, but not fantastic."
- Trial results were presented at the Congress of the European Academy of Neurology in Geneva in June, and Blum flagged uncertainty about whether the drug will work as well in other countries since GBS is triggered by different pathogens depending on region — often Campylobacter jejuni in developing nations and viral infections in developed ones.
Why it matters: For the roughly 8,000 Americans and many more worldwide struck each year by Guillain-Barré syndrome, tanruprubart would offer the first targeted therapy after three decades of only non-targeted plasma exchange and IV immunoglobulin. Its effectiveness outside Bangladesh and the Philippines remains unproven since triggering pathogens differ by region.
