NMPA China Approves Sycume for TED

As China's first and the world's second approved IGF-1R antibody drug, Sycume has ended a 70-year drought of no new treatment option for TED in China. This groundbreaking therapy will redefine the standard of care for TED.

TED is an organ-specific autoimmune disorder closely associated with thyroid disease. It ranks first in the incidence of orbital diseases among adults, particularly prevalent in the age group of 40 to 60 years. The estimated annual incidence of TED is 16 per 100,000 people in women and 2.9 per 100,000 people in men, with a prevalence rate of 0.1-0.3%. TED can cause proptosis, orbital inflammation, diplopia and other clinical manifestations, which significantly impact a patient's appearance, visual function, and quality of life. In severe cases, it can even lead to blindness.

At present, the first-line treatment for moderate-to-severe active TED is intravenous glucocorticoid (IVGC) therapy, although it has limitations, such as inadequate improvement in proptosis and systemic adverse reactions associated with steroid. Second-line treatments include a second course of IVGC, orbital radiotherapy, and other immunosuppressants with suboptimal outcomes. Recent Chinese and international treatment guidelines and joint consensus have recommended the use of IGF-1R-targeted biologics as a second-line treatment for TED. Particularly for patients with significant proptosis or diplopia, IGF-1R-targeted biologics can be considered the first-line treatment in such cases.

Prior to the approval of Sycume, only one IGF-1R antibody drug was approved globally and was not available in China. The cost of IGF-1R antibody oversea is extremely high, costing approximately 3 million RMB per treatment course. As China's first IGF-1R antibody, Sycume offers robust  efficacy and a favorable safety profile, providing a new and accessible treatment option for TED patients, with significant clinical and societal value. Additionally, Sycume adopts a liquid injection formation, which offers strengths in terms of stability, cost, manufacturing process simplicity, and patient compliance.

The approval is based on results from the Phase III registrational study (RESTORE-1) in TED patients, which met its primary endpoint in 2024.. The study showed 85.8% of patients achieved ≥2 mm proptosis reduction at Week 24 with Sycume treatment, accompanied by significant improvements in inflammation and quality of life. The overall safety profile of Sycume was favorable throughout the study. RESTORE-1 results have been presented at premier conferences including World Ophthalmology Congress (WOC), Annual Meeting of Chinese Society of Endocrinology (CSE), and Congress of Chinese Ophthalmological Society (CCOS), generating substantial recognition among experts.

Professor Xianqun Fan, an academician of the Chinese Academy of Engineering, Department of Ophthalmology, Shanghai Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine, TED is the most common orbital disease in adults, and proptosis being its most prominent clinical manifestation. The prolonged course of TED seriously impacts a patient's visual function and appearance, often leading to heavy psychological burden. Current first-line treatment for TED is glucocorticoids, though it has limitations, such as inadequate improvement in proptosis and notable side effects. Biologic agents targeting IGF-1R could to significantly improve proptosis and quality of life in TED patients and are expected to play an important role in its treatment. As an ophthalmologist and the principal investigator of the RESTORE-1 study, I am delighted with the approval of the first new TED drug in China. I hope that this drug will bring high-quality treatment option to TED patients as soon as possible. "