The European Medicines Agency (EMA) has issued a positive recommendation for tolebrutinib, Sanofi’s experimental treatment for multiple sclerosis (MS). This recommendation targets patients with non-relapsing “secondary progressive” MS, a group with significant unmet medical needs. If officially authorized in the coming months, the therapy is expected to be marketed under the brand name Cenrifki.
The EMA’s stance creates a notable regulatory divide, as the US Food and Drug Administration (FDA) rejected the drug earlier this year. The FDA’s primary concern centered on the risk of severe, potentially fatal drug-induced liver injury. While the EMA acknowledged these safety risks, it determined that the clinical benefits—specifically the drug’s ability to delay disability progression—outweighed the potential hazards for this specific patient population.
Tolebrutinib was the centerpiece of Sanofi’s $3.7 billion acquisition of Principia Biopharma in 2020. As a member of the newer class of BTK inhibitors, the drug is designed to cross the blood-brain barrier to address inflammation directly within the central nervous system. Despite setbacks in previous late-stage trials for other forms of MS, this European backing positions Sanofi to finally begin recouping its multi-billion dollar investment in the BTK inhibitor space.