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FDA Grants Breakthrough Therapy Designation to Tolebrutinib for Non-Relapsing Secondary Progressive Multiple Sclerosis

NextEdge Admin

16 Dec 2024

2 min 34 sec

The FDA has granted Breakthrough Therapy designation to tolebrutinib for treating adults with non-relapsing secondary progressive multiple sclerosis (nrSPMS). This decision is based on positive findings from the HERCULES Phase 3 study, which showed a 31% reduction in the risk of 6-month confirmed disability progression (CDP) with tolebrutinib compared to placebo (HR 0.69; 95% CI 0.55-0.88; p=0.0026). Additionally, 10% of participants on tolebrutinib achieved confirmed disability improvement, nearly double that of the placebo group (5%; HR 1.88; 95% CI 1.10-3.21; p=0.021).

Breakthrough Therapy designation is granted to expedite the development of treatments for serious conditions with preliminary evidence of substantial benefit over existing options. No treatments are currently approved for nrSPMS. According to Erik Wallström, MD, PhD, Global Head of Neurology Development at Sanofi, “Tolebrutinib has the potential to address the critical unmet need of delaying disability progression in MS.”

Liver enzyme elevations (>3xULN) were reported in 4.1% of participants on tolebrutinib, with 0.5% experiencing ALT elevations >20xULN, primarily within the first 90 days. Most cases resolved without intervention, and enhanced monitoring has mitigated risks.

Regulatory submissions for tolebrutinib are being finalized for the US and prepared for the EU. Sanofi also anticipates results from the ongoing PERSEUS Phase 3 trial in primary progressive MS in H2 2025.

Tolebrutinib is an investigational, brain-penetrant Bruton’s tyrosine kinase (BTK) inhibitor designed to modulate disease-associated microglia and B lymphocytes. Unlike current treatments that primarily target peripheral immune cells, tolebrutinib acts within the central nervous system, addressing a key driver of disability progression. Its safety and efficacy remain under clinical evaluation.