Two phase 3 studies are currently underway testing riliprubart in people with CIDP as a potential first-in-class treatment.
• The Japanese Ministry of Health, Labour and Welfare (MHLW) has granted orphan drug designation to riliprubart, recognizing its potential to treat chronic inflammatory demyelinating polyneuropathy (CIDP).
• Riliprubart is a monoclonal antibody that selectively inhibits activated C1s in the classical complement pathway.
• This decision aligns with similar designations already granted in the United States and Europe, reflecting global regulatory recognition of its potential in addressing significant unmet medical needs.
• Although therapies for CIDP exist, residual symptoms such as weakness, numbness, and fatigue persist in many patients, leading to long-term morbidity and reduced quality of life.
• An estimated 30% of CIDP patients do not respond to standard treatments, highlighting the need for novel therapeutic options.
• In Japan, approximately 4,000 individuals are currently diagnosed with CIDP.
Comments from Erik Wallstroem: Global Head of Neurology Development, Sanofi
“The orphan drug designation of riliprubart for people living with CIDP in Japan underscores our commitment to applying our deep understanding of the immune system to address rare neurological disorders with significant unmet medical needs. While CIDP therapies exist, many individuals continue to experience debilitating symptoms, including pain, fatigue and weakness. Our ongoing development of riliprubart reflects our dedication to challenging the status quo in neurology with the goal of improving people’s lives.”
• 76-week data from a Phase 2 study of riliprubart were presented at the Peripheral Nerve Society meeting (Edinburgh, UK; May 17–20, 2025).
• Results indicate long-term, sustained efficacy and safety in a broad population of patients with chronic inflammatory demyelinating polyneuropathy (CIDP).
• ‘Riliprubart is now being evaluated in two Phase 3 trials:
MOBILIZE: In patient’s refractory to standard of care
ClinicalTrials.gov Identifier: NCT06290128
VITALIZE: In IVIg-treated patients
ClinicalTrials.gov Identifier: NCT06290141
• The data support riliprubart’s potential as a long-term treatment option for CIDP.
• SAR445088 (riliprubart) is an IgG4 humanized monoclonal antibody that selectively inhibits activated C1s in the classical complement pathway of the innate immune system.
• By blocking C1s, riliprubart has the potential to inhibit key inflammatory mechanisms that drive demyelination and axonal damage in CIDP.
• Riliprubart is currently under clinical investigation, and its safety and efficacy have not been evaluated by any regulatory authority. For more information on riliprubart clinical studies, please visit www.clinicaltrials.gov.
• CIDP is a rare neurological condition that causes progressive weakness and sensory impairment in the arms and legs.
• CIDP occurs when the body’s immune system attacks the myelin sheaths around nerve cells in the peripheral nervous system.
• Timely diagnosis of CIDP is important because it allows for appropriate treatment, which is essential to preventing long-term disability. However, despite available therapies, many individuals are left with residual symptoms, including weakness, numbness, and fatigue that can lead to long-term morbidity and diminished quality-of-life.
• Approximately 30% of people with CIDP do not respond to standard therapies. In people with CIDP who do respond, about 70% of the response is considered incomplete. Less than one-third of people with CIDP remain in remission without continued therapy.
• Sanofi is an R&D driven, AI-powered biopharma company committed to improving people’s lives and delivering compelling growth.
• We apply our deep understanding of the immune system to invent medicines and vaccines that treat and protect millions of people around the world, with an innovative pipeline that could benefit millions more.
• Our team is guided by one purpose: we chase the miracles of science to improve people’s lives; this inspires us to drive progress and deliver positive impact for our people and the communities we serve, by addressing the most urgent healthcare, environmental, and societal challenges of our time.
Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY
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