Riliprubart Gets Orphan Drug Status in Japan for CIDP Treatment

Riliprubart Received Orphan Drug Designation in Japan

Image Credits: Western Slope Now

Announcement

The Japanese Ministry of Health, Labour and Welfare appointed riliprubart as an orphan drug designation. Riliprubart is a monoclonal antibody suppressing active C1s in the classical complement for individuals with chronic inflammatory demyelinating polyneuropathy (CIDP). Though several therapies have been introduced, many CIDP patients contain residual symptoms such as numbness, fatigue, and weakness, leading quality of life and long-term morbidity. Around 30% of people with CIDP don’t react to standard therapies. The MHLW awards orphan drug designation to those medicines that identify rare medical diseases with unmet medical needs. There are around 4,000 individuals admitted with CIDP in Japan. The two phase 3 studies are in the testing process of riliprubart in individuals with CIDP to receive acceptance as a first-in-class treatment. MHLW’s decision is an addition to the in-common designation in Europe and the US.

Riliprubart

Riliprubart (SAR445088) is an IgG4 humanized monoclonal antibody that specifically hinders activated C1s in the immune system. After stopping C1s, Riliprubart unlocks its potential to hinder key inflammatory mechanisms that cause axonal and demyelination damage in CIDP. This drug is under clinical investigation, as its efficacy and safety have not been evaluated by any regulatory authority. Additional details and updates on ongoing studies on riliprubart are available on www.clinicaltrials.gov.

Study

A study of its long-term 76-week sustained efficacy with safety covering riliprubart’s phase 2 study conducted bat the Peripheral Nerve Society, meeting in Edinburgh, UK on 17TH MAY 2025. The findings of the study reveal the potential and its long-term benefit of riliprubart in individuals with CIDP. Riliprubart has recently been tested in two separate phase 3 studies, one is VITALIZE in IVIg-treated patients (clinical study identifier: NCT06290141) and the other is MOBILIZE in patients refractory to standard of care (clinical study identifier: NCT06290128). The profound study will ensure better and effective outcomes for patients and providers as well. Regulatory recognition is a benefit for further discoveries and development in medicines and therapies, accelerating the healthcare market.

Views and Statements

Global Head of Neurology Development, MD, PHd, Sanofi, Erik Wallstroem said, “The official entry of Riliprubart meets our commitment to consider an understanding of the immune system, identifying rare neurological disorders with required unmet medical needs. Despite, existence of CIDP therapies, people experience symptoms such as weakness, pain, and fatigue. Companies, current development is in progress and fueling the dedication to challenge the status Quo in neurology.”