By VETTAPHARMA reporter – Derek Roche: The European Commission has approved Sanofi’s Cenrifki (tolebrutinib) for the treatment of adults with secondary progressive multiple sclerosis (SPMS) without relapses in the previous two years, making it the first disability-targeting medicine approved in the European Union for this patient population. The approval addresses a major unmet need by targeting disability progression rather than relapse activity in progressive multiple sclerosis.
The approval follows a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) and is supported by results from the pivotal Phase 3 HERCULES trial (NCT04411641) in non-relapsing SPMS, together with supportive evidence from the Phase 3 GEMINI 1 (NCT04410978) and GEMINI 2 (NCT04410991) studies in relapsing multiple sclerosis. In HERCULES, Cenrifki significantly delayed the onset of disability progression in patients with non-relapsing SPMS.
Cenrifki is an oral, brain-penetrant Bruton’s tyrosine kinase (BTK) inhibitor designed to target smoldering neuroinflammation, a key biological driver of disability accumulation in multiple sclerosis. According to Sanofi, it is the first therapy approved in the EU specifically designed to target the underlying process responsible for disability progression in adults with SPMS without relapses during the previous two years.
The safety profile was consistent across the clinical development program. The most frequently reported adverse events were COVID-19 and upper respiratory tract infections. Significant liver enzyme elevations were also observed, and drug-induced liver injury (DILI) has been identified as an important safety risk. The company emphasized that strict liver monitoring and prompt management of liver enzyme elevations are essential to mitigate this risk.
Sanofi plans to launch Cenrifki commercially in Germany later this year, supported by a comprehensive Risk Management Program and Patient Support Program. The medicine has also received approvals in Australia and the United Arab Emirates for non-relapsing SPMS indications.
Quick FAQs
1. What Is Cenrifki?
Cenrifki (tolebrutinib) is an oral, brain-penetrant Bruton’s tyrosine kinase (BTK) inhibitor designed to target smoldering neuroinflammation that contributes to disability progression in multiple sclerosis.
2. What Happened?
The European Commission approved Sanofi’s Cenrifki for adults with secondary progressive multiple sclerosis without relapses in the previous two years, making it the first disability-targeting medicine approved in the EU for this patient population.
3. What Supported The Approval?
The approval was based on the Phase 3 HERCULES (NCT04411641) study in non-relapsing SPMS, with supportive evidence from the Phase 3 GEMINI 1 (NCT04410978) and GEMINI 2 (NCT04410991) studies in relapsing multiple sclerosis.
4. What Is Secondary Progressive Multiple Sclerosis?
Secondary progressive multiple sclerosis is a stage of multiple sclerosis characterized by continuous accumulation of disability, including impaired mobility, fatigue, cognitive decline, and loss of independence, often without relapses.
5. What Were The Key Findings?
The HERCULES Phase 3 trial demonstrated that Cenrifki significantly delayed the onset of disability progression in patients with non-relapsing SPMS. The safety profile was consistent across studies, with COVID-19 and upper respiratory tract infections being the most common adverse events, while liver monitoring remains essential because of the risk of drug-induced liver injury.
6. Why Is This Important?
Cenrifki is the first therapy approved in the European Union to specifically target disability progression in adults with non-relapsing SPMS, addressing one of the most significant unmet needs in multiple sclerosis care.
