Nusinersen effectiveness in teens with Type II/III 5q-spinal muscular atrophy: 3rd interim analysis from a Chinese registry

Topic:

Clinical Trials

Poster Number: 203 M

Author(s):

Rong Luo, West China Second University Hospital, Sichuan University, Xiaoli Yao, The First Affiliated Hospital, Sun Yat-sen University, Jing Peng, Xiangya Hospital Central South University, Xiuxia Wang, The Second Hospital of Hebei Medical University, Hanbing Lu, Xuzhou Central Hospital, Jianmin Liang, The First Hospital of Jilin University, Lin Yang, The Second Affiliated Hospital of Xi’An Jiaotong University, Yiyan Ruan, Maternity and Child Health Care of Guangxi Zhuang Autonomous Region, Ruifeng Jin, Children’s Hospital Affiliated to Shandong University, Jianmin Zhong, Jiangxi Provincial Children’s Hospital, Xiaoli Zhang, The Third Affiliated Hospital of Zhengzhou University, Xufeng Luo, West China Second University Hospital, Liwen Wu, Hunan Children’s Hospital, Jihua Wu, Anhui Children’s Hospital, Siqi Hong, MD, Children’s Hospital of Chongqing Medical University, Shanshan Mao, Children’s Hospital Zhejiang University School of Medicine, Hua Wang, Shengjing Hospital of China Medical University, Dan Sun, Wuhan Children’s Hospital, Tongji Medical College Huazhong University of Science & Technology, Jun Hu, Fujian Medical University Union Hospital, Zhe Tao, Dalian Women and Children’s Medical Group, Guanglu Yang, The Affiliated Hospital of Inner Mongolia Medical University, Hui Xiong, MD, PhD, Children’s Medical Center, Peking University First Hospital, Lanlan Meng, Biogen Biotechnology (Shanghai) Co., Ltd, Li Zhang, MD, Biogen Biotechnology (Shanghai) Co., Ltd, Junli Zhu, IQVIA Solutions Enterprise Management Consulting (Shanghai) Co., Ltd, Yi Wang, Children’s Hospital of Fudan University

Yi Wang is corresponding author.

Background and objective: To assess the long-term effectiveness of nusinersen in children aged ≥ 10 years with Type II/III 5q-spinal muscular atrophy (SMA).

Methods: A multicenter, longitudinal registry was established to collect data retrospectively and prospectively from children with 5q-SMA across 25 sites in China. Among those who initiated nusinersen treatment at age ≥ 10 years, baseline demographic and clinical characteristic were evaluated. Motor function was evaluated at 6, 14, 26, 38 months following treatment initiation using the Hammersmith Functional Motor Scale Expanded (HFMSE), Revised Upper Limb Module (RULM), and 6-Minute Walk Test (6-MWT).

Results: As of June 11th, 2025, a total of 126 participants with Type II (n=43) and III (n=83) SMA were analyzed. At baseline, scoliosis occurred in all Type II and 54 (65.1%) Type III participants, while contractures mainly involved the knee (55.0% vs 17.5%) and ankle (47.5% vs 16.3%). Baseline mean (standard deviation, SD) scores were 7.15 (10.014) for HFMSE and 13.00 (8.195) for RULM in Type II participants, and 38.07 (17.662) and 30.05 (7.007), respectively, in Type III participants.
For participants with Type II SMA, the mean (SD) HFMSE changes from baseline to 6, 14, 26, 38 months were 5.47 (6.530), 3.40 (3.921), 2.00 (4.870), and 0.75 (6.652), respectively, while the corresponding RULM changes were 2.47 (5.316), 3.30 (6.093), 4.83 (5.981), and -1.00 (5.657).
For participants with Type III SMA, the mean (SD) HFMSE changes from baseline to 6, 14, 26, 38 months were 3.06 (5.101), 3.07 (5.267), 4.50 (4.274), and 7.00 (5.686). The corresponding RULM changes were 1.45 (2.353), 0.67 (4.721), 2.29 (3.518), and 3.33 (1.155). The 6-MWT changes were 22.91 (62.533), 39.94 (64.464), 98.03 (50.759), and 78.55 (43.378).

Conclusion: Nusinersen treatment resulted in maintained motor performance and functional stability in Chinese teenagers with Type II/III SMA, underscoring the continued effectiveness of nusinersen in older pediatric populations.

Disclaimer: This study was sponsored by Biogen. Writing and editorial support for the preparation of this abstract was provided by IQVIA: funding was provided by Biogen. Lanlan MENG and Li ZHANG are employees of Biogen and may hold stock in Biogen. Junli ZHU is an employee of IQVIA.

Key words: Spinal muscular atrophy; Nusinersen; Pediatric; Registry; Teenager