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6-K: Novartis Intrathecal Onasemnogene Abeparvovec Phase Iii Study Meets Primary Endpoint in Children and Young Adults with SMA

6-K: Novartis Intrathecal Onasemnogene Abeparvovec Phase Iii Study Meets Primary Endpoint in Children and Young Adults with SMA

6-K:诺华鞘内奥那西莫金 Abeparvovec Iii 期研究达到 SMA 儿童和青少年患者的主要终点
美股SEC公告 ·  2024/12/30 11:10

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Novartis announced positive topline results from the Phase III STEER study evaluating intrathecal onasemnogene abeparvovec (OAV101 IT) in treatment-naïve SMA Type 2 patients aged 2-18 years. The study met its primary endpoint, demonstrating increased Hammersmith Functional Motor Scale - Expanded (HFMSE) scores compared to sham controls, indicating improved motor function in patients.The safety profile was favorable, with similar adverse events between treatment and control arms. The most common side effects included upper respiratory tract infection, pyrexia, and vomiting. The study involved over 100 patients who were able to sit but had never walked independently.Novartis plans to share results with regulatory agencies, including the FDA, in 2025 to pursue approval. The STEER results build on the previous Phase I/II STRONG study, which showed clinically meaningful HFMSE score increases in one year. This gene therapy represents a potential one-time treatment option for SMA patients who currently rely on chronic treatments.
Novartis announced positive topline results from the Phase III STEER study evaluating intrathecal onasemnogene abeparvovec (OAV101 IT) in treatment-naïve SMA Type 2 patients aged 2-18 years. The study met its primary endpoint, demonstrating increased Hammersmith Functional Motor Scale - Expanded (HFMSE) scores compared to sham controls, indicating improved motor function in patients.The safety profile was favorable, with similar adverse events between treatment and control arms. The most common side effects included upper respiratory tract infection, pyrexia, and vomiting. The study involved over 100 patients who were able to sit but had never walked independently.Novartis plans to share results with regulatory agencies, including the FDA, in 2025 to pursue approval. The STEER results build on the previous Phase I/II STRONG study, which showed clinically meaningful HFMSE score increases in one year. This gene therapy represents a potential one-time treatment option for SMA patients who currently rely on chronic treatments.
诺华宣布了III期STEER研究的积极顶线结果,该研究评估了在2至18岁未经治疗的SMA 2型患者中使用脊髓内注射的onasemnogene abeparvovec (OAV101 IT)。该研究达到了主要终点,显示治疗组的Hammersmith功能运动量表扩展版(HFMSE)评分相比假治疗组有所增加,表明患者的运动功能得到改善。安全性方面情况良好,治疗组和对照组的不良事件类似。最常见的副作用包括上呼吸道感染、发热和呕吐。该研究涉及了100多名能够坐但从未独立行走的患者。诺华计划在2025年与包括FDA在内的监管机构分享结果,以争取批准。STEER研究结果建立在之前的I/II期STRONG研究的基础上,该研究显示HFMSE评分在一年内有临床意义的提高。这种基因治疗代表了一种潜在的单次治疗选择,适用于目前依赖长期治疗的SMA患者。
诺华宣布了III期STEER研究的积极顶线结果,该研究评估了在2至18岁未经治疗的SMA 2型患者中使用脊髓内注射的onasemnogene abeparvovec (OAV101 IT)。该研究达到了主要终点,显示治疗组的Hammersmith功能运动量表扩展版(HFMSE)评分相比假治疗组有所增加,表明患者的运动功能得到改善。安全性方面情况良好,治疗组和对照组的不良事件类似。最常见的副作用包括上呼吸道感染、发热和呕吐。该研究涉及了100多名能够坐但从未独立行走的患者。诺华计划在2025年与包括FDA在内的监管机构分享结果,以争取批准。STEER研究结果建立在之前的I/II期STRONG研究的基础上,该研究显示HFMSE评分在一年内有临床意义的提高。这种基因治疗代表了一种潜在的单次治疗选择,适用于目前依赖长期治疗的SMA患者。
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