FDA Delays Decision on Tolebrutinib for Non-Relapsing Secondary Progressive MS
The Food and Drug Administration (FDA) has extended its review period for tolebrutinib (Sanofi, Bridgewater, NJ), moving the Prescription Drug User Fee Act (PDUFA) decision date from September 28 to December 28, 2025. Tolebrutinib is an oral, brain-penetrant Bruton's tyrosine kinase (BTK) inhibitor under review for the treatment of adults with non-relapsing, secondary progressive multiple sclerosis (nrSPMS). The therapy is designed to slow disability accumulation independent of relapses.
The delay follows Sanofi’s submission of additional analyses during the review process, which the FDA classified as a major amendment to the new drug application (NDA). As a result, the Agency extended the review period by 3 months to allow for full evaluation of the updated package. The NDA is supported by data from the phase 3 HERCULES clinical trial (NCT04411641), evaluating tolebrutinib treatment for people with nrSPMS, and the GEMINI 1 (NCT04410978) and 2 (NCT04410991) studies, evaluating treatment for people with relapsing multiple sclerosis (MS). An additional phase 3 trial, PERSEUS (NCT04458051), evaluating tolebrutinib treatment for people with primary progressive MS, is ongoing, with results expected later in 2025.
If approved, tolebrutinib would become the first BTK inhibitor available for nrSPMS, targeting innate immune activity within the central nervous system, an area not directly addressed by current MS therapies.
Sources: Samorodnitsky D. FDA action date for Sanofi’s oral multiple sclerosis drug bumped to December. Biospace.com. Published September 22, 2025. Accessed September 22, 2025. https://www.biospace.com/fda/fda-action-date-for-sanofis-oral-multiple-sclerosis-drug-bumped-to-december
Sanofi. Press release: update on the US regulatory review of tolebrutinib in non-relapsing, secondary progressive multiple sclerosis. Sanofi.com. Published September 22, 2025. Accessed September 22, 2025. https://www.sanofi.com/en/media-room/press-releases/2025/2025-09-22-05-00-00-3153624