| 商品名称 | Libmeldy |
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| 适用类别 | Human |
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| 治疗领域 | Leukodystrophy, Metachromatic |
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| 通用名/非专利名称 | autologous CD34+ cells encoding ARSA gene |
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| 活性成分 | atidarsagene autotemcel |
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| 产品号 | EMEA/H/C/005321 |
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| 患者安全信息 | No |
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| 许可状态 | Authorised |
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| ATC编码 | N07 |
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| 是否额外监管 | Yes |
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| 是否仿制药或hybrid药物 | No |
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| 是否生物类似药 | No |
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| 是否附条件批准 | No |
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| 是否特殊情形 | No |
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| 是否加速审评 | No |
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| 是否罕用药 | Yes |
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| 上市许可日期 | 2020/12/17 |
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| 上市许可开发者/申请人/持有人 | Orchard Therapeutics (Netherlands) B.V. |
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| 人用药物治疗学分组 | Other nervous system drugs |
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| 兽用药物治疗学分组 | |
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| 审评意见日期 | 2020/10/15 |
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| 欧盟委员会决定日期 | 2025/12/15 |
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| 修订号 | 9 |
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| 治疗适应症 | Libmeldy is indicated for the treatment of metachromatic leukodystrophy (MLD) characterized by biallelic mutations in the arysulfatase A (ARSA) gene leading to a reduction of the ARSA enzymatic activity: in children with late infantile or early juvenile forms, without clinical manifestations of the disease, in children with the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. |
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| 适用物种 | |
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| 兽用药物ATC编码 | |
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| 首次发布日期 | 2020/12/22 |
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| 最后更新日期 | 2025/12/16 |
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| 产品说明书 | https://www.ema.europa.eu/en/documents/product-information/libmeldy-epar-product-information_en.pdf |
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| 公共评估报告 | https://www.ema.europa.eu/en/medicines/human/EPAR/libmeldy |
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