FDA grants breakthrough therapy designation to tolebrutinib for MS

The FDA granted breakthrough therapy designation to an oral, investigational, brain-penetrant and bioactive Bruton’s tyrosine kinase inhibitor for adults with non-relapsing secondary progressive MS.

According to a release from drug manufacturer Sanofi, the ruling was made after positive results were reported from the phase 3 HERCULES clinical trial, in which tolebrutinib delayed onset of 6-month confirmed disability progression by 31% compared placebo. In addition, treatment with tolebrutinib led to almost double the number of study participants who experienced confirmed disability improvement vs. those given placebo.

Sanofi in the release it is preparing regulatory submissions for the novel therapeutic in both the United States and European Union. Tolebrutinib is also being investigated in the ongoing phase 3 PERSEUS study in adults with primary progressive MS, with study results anticipated by the second half of 2025.

“This breakthrough therapy designation demonstrates the potential for tolebrutinib to delay disability progression, a critical unmet need for people living with MS,” Erik Wallström, MD, PhD, global head of neurology development, Sanofi, in the release. “We look forward to working with the FDA during the regulatory review of this first of its kind medicine in non-relapsing secondary progressive MS where there are currently no approved treatments available.”