| 商品名称 | Waskyra |
|---|---|
| 适用类别 | Human |
| 治疗领域 | Wiskott-Aldrich Syndrome |
| 通用名/非专利名称 | etuvetidigene autotemcel |
| 活性成分 | genetically modified autologous CD34+ cell enriched population that contains haematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human Wiskott-Aldrich Syndrome (WAS) gene |
| 产品号 | EMEA/H/C/006525 |
| 患者安全信息 | No |
| 许可状态 | Authorised |
| ATC编码 | B06 |
| 是否额外监管 | Yes |
| 是否仿制药或hybrid药物 | No |
| 是否生物类似药 | No |
| 是否附条件批准 | No |
| 是否特殊情形 | No |
| 是否加速审评 | No |
| 是否罕用药 | Yes |
| 上市许可日期 | 2026/01/09 |
| 上市许可开发者/申请人/持有人 | Fondazione Telethon ETS |
| 人用药物治疗学分组 | Other hematological agents |
| 兽用药物治疗学分组 | |
| 审评意见日期 | 2025/11/13 |
| 欧盟委员会决定日期 | 2026/01/09 |
| 修订号 | |
| 治疗适应症 | Waskyra is indicated for the treatment of patients aged 6 months and older with Wiskott-Aldrich Syndrome (WAS) who have a mutation in the WAS gene for whom haematopoietic stem cell (HSC) transplantation is appropriate and no suitable human leukocyte antigen (HLA)-matched related haematopoietic stem cell donor is available. |
| 适用物种 | |
| 兽用药物ATC编码 | |
| 首次发布日期 | 2025/11/14 |
| 最后更新日期 | 2026/03/05 |
| 产品说明书 | https://www.ema.europa.eu/en/documents/product-information/waskyra-epar-product-information_en.pdf |
| 公共评估报告 | https://www.ema.europa.eu/en/medicines/human/EPAR/waskyra |