On June 16, 2025, RemeGen Co., Ltd. („RemeGen“, stock symbols: 688331.SH/09995.HK) announced that telitacicept (RC18; brand name: ??®) has received Orphan Drug Designation (ODD) from European Medicines Agency (EMA) for the treatment of Myasthenia Gravis (MG), marking a key milestone achieved in its global development. Telitacicept now is the first dual-target biologic drug for MG with ODD from both FDA and EMA worldwide.
The designation, granted based on telitacicept’s significant benefits in treating the life-threatening rare disease of MG, will provide advantages to this novel agent on protocol assistance, regulatory fee reductions/waivers and up to 10 years of market exclusivity, thereby accelerating the clinical development, registration and drug approval process in Europe and its availability among MG patients.
MG is an acquired antibody-mediated rare autoimmune disorder affecting the neuromuscular junction. According to the Myasthenia Gravis Foundation of America (MGFA) and various studies, its global prevalence is estimated to be 15-25 per 100,000, meeting EMA’s definition of the rare disease (the prevalence of less than 5 in 10,000 in the EU). Though the current treatment options (including cholinesterase inhibitors, glucocorticoids, immunosuppressants, intravenous immunoglobulins, plasma exchange, and targeted biological agents) may help to manage symptoms, many patients still suffer from poor response, drug intolerance and disease relapse, highlighting a significant unmet clinical need.
