Sanofi’s Riliprubart granted orphan drug designation for chronic inflammatory demyelinating polyneuropathy in Japan

Sanofi announces that the Ministry of Health, Labour and Welfare in Japan has granted orphan drug designation to riliprubart. Despite available therapies, many CIDP patients 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. Note that the MHLW grants orphan drug designation to medicines that address rare medical diseases or conditions with unmet medical needs.

Erik Wallstroem, MD, PhD
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.”

The safety data from riliprubart’s phase 2 study were presented at the Peripheral Nerve Society meeting in Edinburgh, UK on May 17-20, 2025. Findings suggest a potential sustained long-term benefit provided by riliprubart across a broad spectrum of participants with CIDP. Riliprubart is currently being tested in two separate phase 3 studies: MOBILIZE in patients refractory to standard of care and VITALIZE in IVIg-treated patients.

---

---

Related Topics and Keywords

Chronic Inflammatory Demyelinating Polyneuropathy, chronic inflammatory demyelinating polyneuropathy chronic inflammatory demyelinating polyneuropathy, Riliprubart