EMA Grants Orphan Drug Status to RemeGen's Telitacicept for Myasthenia Gravis

YANTAI, China, June 17, 2025 – RemeGen Co., Ltd., a prominent biopharmaceutical company, has reached a significant milestone in its global expansion with the announcement that its innovative drug, telitacicept (RC18), marketed under the brand name 泰爱®, has secured Orphan Drug Designation (ODD) from the European Medicines Agency (EMA). This accomplishment marks telitacicept as the first dual-target biologic drug for Myasthenia Gravis (MG) to receive this recognition from both the U.S. Food and Drug Administration (FDA) and the EMA.

The ODD status is an important step for telitacicept, as it is based on the drug’s notable benefits in addressing Myasthenia Gravis, a rare and life-threatening autoimmune disease. This designation provides several advantages, including protocol assistance, reductions or waivers of regulatory fees, and up to a decade of market exclusivity. Such benefits are crucial for accelerating the clinical development, registration, and approval processes in Europe, ultimately facilitating broader access for MG patients.

Myasthenia Gravis is an acquired autoimmune disorder characterized by the body's immune system attacking the neuromuscular junction, leading to muscle weakness. According to the Myasthenia Gravis Foundation of America (MGFA) and other research, the global prevalence of this condition ranges from 15 to 25 per 100,000 individuals, meeting the EMA's criteria for a rare disease, which is defined by a prevalence of less than 5 in 10,000 within the European Union. While existing treatments—including cholinesterase inhibitors, glucocorticoids, immunosuppressants, intravenous immunoglobulins, plasma exchange, and targeted biological agents—can alleviate symptoms, many patients continue to experience inadequate responses, drug intolerance, and disease relapses, underscoring an urgent need for more effective therapies.

Telitacicept stands out as the world’s first approved fusion protein drug that targets both BLyS and APRIL pathways, making it a groundbreaking advancement in MG treatment. The disease progresses as pathological B cells generate autoantibodies that attack proteins in the neuromuscular junction, such as the acetylcholine receptor. Telitacicept intervenes by inhibiting both BLyS and APRIL signaling pathways, effectively suppressing the activation of these harmful B cells and reducing the production of these damaging autoantibodies. This dual-action approach aims to address the root cause of MG progression.

In China, telitacicept was approved for marketing as recently as May. Its phase III clinical trials have shown promising results, with 98.1% of participants achieving an improvement of 3 or more points in the myasthenia gravis-activities of daily living (MG-ADL) score after a 24-week treatment period, compared to just 12.0% in the placebo group. Furthermore, 87% of participants reported a 5-point or greater improvement in the quantitative myasthenia gravis (QMG) score, in contrast to 16.0% in the placebo group. These outcomes demonstrate a statistically significant difference in efficacy compared to the placebo, alongside a manageable safety profile.

The recognition of telitacicept by the EMA through the ODD is a testament to its innovative mechanism and its potential to transform the management of MG. RemeGen is committed to advancing the global multi-center phase III clinical trials of telitacicept for patients with MG, aiming to bring this pioneering treatment to patients worldwide.