Efficacy and safety of tolebrutinib in multiple sclerosis: a systematic review and meta-analysis

Tolebrutinib is an oral, brain-penetrant Bruton's tyrosine kinase (BTK) inhibitor being investigated for multiple sclerosis (MS). Early data point to reduced relapse rates and radiological disease activity, along with a reassuring safety profile. This systematic review and meta-analysis bring together the available evidence on its clinical efficacy and adverse events.

We searched PubMed, Web of Science, and Scopus through February 2026 using predefined eligibility criteria. Data were extracted on efficacy outcomes (disability progression and improvement, MRI activity, and brain volume change) and safety outcomes (any adverse events, serious adverse events, adverse events leading to discontinuation, headache, ALT elevation, and death). Continuous outcomes were pooled as means using reported means and standard deviations; proportional outcomes were pooled from event and total counts. Analyses were run in R using random-effects models, with data unsuitable for pooling described narratively.