After a series of regulatory and clinical setbacks for Sanofi’s tolebrutinib, the French pharma has scored a victory for the multiple sclerosis (MS) candidate. Thursday, Europe’s Committee for Medicinal Products for Human Use (CHMP) recommended its approval for patients with secondary progressive MS (SPMS) who have not had relapses in the last two years. The CHMP cited the benefits of tolebrutinib, including a 31% reduction in disability progression and a 38% reduction in new or enlarging lesions per year compared with patients on placebo. In Europe, the drug will carry the commercial moniker Cenrifki.
Last year, Sanofi announced two regulatory delays in the United States before the FDA rejected the treatment in the same indication in December, citing efficacy and safety issues. In its complete response letter (CRL), the agency said that “a favorable benefit-risk profile could not be established for any patient subpopulation” for tolebrutinib.
Also in December of last year, Sanofi disclosed the flop of a phase 3 trial assessing tolebrutinib as a treatment for primary progressive MS (PPMS), ending its hopes of gaining approval in the indication. That came on top of failures for tolebrutinib in two other late-stage MS trials in 2024.
Sanofi picked up the BTK inhibitor as part of its $3.7 billion buyout of Principia Biopharma in 2020. Holding tolebrutinib back in the U.S. has been the risk of drug-induced liver injury (DILI), which can’t be adequately managed by Sanofi’s proposed risk evaluation and mitigation strategy (REMS), according to the FDA in its most recent CRL.
