法规知识库 · FDA 药品生命周期
首次正式批准
2020-08-12
原始申请分类
TYPE 1 · 新分子实体（NME）
当前持有企业
NIPPON SHINYAKU
最新审批记录
2021-03-11
药品申请
1
NDA212154
产品/规格
1
当前快照
审批记录
2
1 次 SUPPL
文件记录
5
索引 5
NDA212154 · 处方药
规格与剂型
靶点与作用机制
靶点
DMD, dystrophin
作用机制（MoA）
ASO (PMO), exon 53 skipping
来源 ORIG:1
当前获批适应症（说明书）
VILTEPSO（维托拉生）是一种反义寡核苷酸，适用于治疗经确认存在DMD基因突变且适合外显子53跳跃的杜氏肌营养不良（DMD）患者；该加速批准基于骨骼肌肌营养不良蛋白（dystrophin）生成增加。
产品号 001 · VILTEPSO · 250MG/5ML (50MG/ML) · SOLUTION;INTRAVENOUS
监管信息
2021
03/11
批准信：Prior Approval；说明书安全性相关更新
批准信原文要点
This Prior Approval supplemental new drug application provides for changes to the language in the Prescribing Information in Dosage and Administration (Subsection 2.1) and Warnings and Precautions (Subsection 5.1) for timing of urine samples because of a potential test interfe…
Labeling-Package Insert（PI）
2020
08/12
批准信：加速批准 Viltepso（viltolarsen）注射液，用于确诊DMD基因突变且适合53号外显子跳跃的杜氏肌营养不良（DMD）患者；并授予罕见儿科疾病优先审评券。
批准信原文要点
This new drug application provides for the use of Viltepso (viltolarsen) injection for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. It is approved under the provisions of accelerated approval regulations (21 CFR 314.500). We also inform you that you have been granted a rare pediatric disease priority review voucher.
本申报适应症：适用于杜氏肌营养不良（DMD）患者，其DMD基因存在确认的、可适合外显子53跳跃的突变；基于新生截短肌营养不良蛋白的增加获得加速批准（80 mg/kg，每周一次静脉输注）。
疾病背景介绍：DMD在5至24岁男性中的患病率估计为每万人1.4例；约8%的患者存在可通过外显子53跳跃纠正的缺失突变。
现有治疗手段：现有SOC治疗：地氟可特（Deflazazort，Emflaza），一种糖皮质激素，已获DMD完全批准，但缓解率有限；依替利森（eteplirsen，外显子51跳跃）和戈洛迪森（golodirsen，外显子53跳跃）均通过加速批准途径获批，临床获益尚未确认。
CMC：Sterile, preservative-free solution of viltolarsen (250 mg/mL) in 0.9% saline for IV infusion over 60 minutes; doses <5000 mg (100 mL) diluted with 0.9% saline to 100 mL. Specifications include appropriate tests for oligonucleotides; elemental impurities testing omission supported by risk assessment. Up to 18 months long-term stability data (5°C ± 3°C) and 6 months accelerated (25°C/60%RH); proposed 18-month shelf life at 2°C to 8°C acceptable. Commercial manufacturing process and in-process controls deemed adequate; all manufacturing facilities acceptable; no outstanding OPQ issues.
来源：212154Orig1s000SumR.pdf · 打开摘录来源 ↗