Tolebrutinib regulatory submission accepted for priority review in the US for patients with multiple sclerosis
March 25, 2025 – Clinical Trials, Drug Discovery, Other, Pharmaceutical – Sanofi, clinical trials, multiple sclerosis, priority review, smouldering neuroinflammation
- If approved, tolebrutinib would be the first and only brain-penetrant BTK inhibitor to both treat non-relapsing secondary progressive multiple sclerosis (MS) and slow disability accumulation independent of relapse activity
- Tolebrutinib has the potential to be the first therapy to target smoldering neuroinflammation, a key driver of disability accumulation in MS
- Tolebrutinib was granted breakthrough therapy designation by the FDA based on positive results from the HERCULES phase 3 study in adults with non-relapsing secondary progressive MS.
25 March 2025 — Paris, France — The US Food and Drug Administration (FDA) is evaluating under priority review the regulatory submission of tolebrutinib to treat non-relapsing secondary progressive multiple sclerosis (nrSPMS) and to slow disability accumulation independent of relapse activity in adult patients. The target action date for the FDA decision is September 28, 2025. A regulatory submission is also under review in the EU.
The regulatory submissions in the US and the EU are supported by the results from the phase 3 studies HERCULES in nrSPMS and GEMINI 1 and 2 in relapsing MS (RMS). The findings from these studies, as well as additional clinical and preclinical studies, support the differentiated mechanism of tolebrutinib to target disability progression independent of relapse activity, and the scientific hypothesis that smoldering neuroinflammation represents a key inflammatory process in MS and is a critical driver of disability accumulation.
Erik Wallström, MD, PhD, Global Head of Neurology Development, “The totality of data across our clinical program validates our scientific understanding of smoldering neuroinflammation as a distinct inflammatory process in MS. People living with non-relapsing secondary progressive multiple sclerosis or who experience disability independent of relapse activity suffer from disability that worsens over time due to persistent inflammation in the brain, known as smoldering neuroinflammation, which is the primary driver of disability. The demonstrated ability of tolebrutinib to delay disability by targeting underlying drivers of the disease represents a potential paradigm shift in treating these patients.”
Tolebrutinib is currently under clinical investigation, and its safety and efficacy have not been evaluated by any regulatory authority. The PERSEUS phase 3 study of tolebrutinib in patients with primary progressive MS is currently ongoing with study results anticipated in H2 2025.
About Sanofi
We are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people’s lives. Our team, across the world, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the center of our ambitions. Visit: www.sanofi.com/en.
