FDA Panel Backs Zevra's Arimoclomol For Rare Genetic Disorder, Analyst Highlights Balanced Portfolio Of Marketed Products And Pipeline Assets Focused On Rare Diseases
FDA Panel Backs Zevra's Arimoclomol For Rare Genetic Disorder, Analyst Highlights Balanced Portfolio Of Marketed Products And Pipeline Assets Focused On Rare Diseases
Friday, the FDA's Genetic Metabolic Diseases Advisory Committee (GeMDAC) voted in favor of Zevra Therapeutics, Inc.'s (NASDAQ:ZVRA) arimoclomol.
FDA的基因代谢病顾问委员会(GeMDAC)在星期五投票通过了Zevra Therapeutics, Inc.(NASDAQ:ZVRA)的arimoclomol。
The committee voted favorably (11 yes, 5 no) that the data supports that arimoclomol is effective in treating patients with Niemann-Pick disease type C (NPC), a rare progressive genetic disorder characterized by an inability of the body to transport cholesterol and other fatty substances (lipids) inside of cells.
委员会投票赞成(11票赞成, 5票反对), 认为数据支持arimoclomol有效治疗尼曼-皮克病C型(NPC)患者, 这是一种罕见的进行性遗传性疾病, 其特点是身体无法将胆固醇和其他脂质物质运输到细胞内。
The committee's recommendation will be considered by the FDA as it completes its independent review of the arimoclomol NDA; however, the feedback from the GeMDAC is not binding upon the agency.
FDA将在完成对arimoclomol NDA的独立审查时考虑委员会的建议, 但GeMDAC的反馈不对该机构具有约束力。
The arimoclomol NDA has been assigned a September 21 Prescription Drug User Fee Act (PDUFA) action date.
arimoclomol NDA已被指定为9月21日处方药用户费用法案(PDUFA)的行动日期。
In 2021, the FDA issued a complete response letter to arimoclomol as a treatment option for Niemann-Pick disease type C.
2021年，FDA发出完整的答复信，称arimoclomol不是治疗Niemann-Pick病C型的选择。
The CRL asked for additional qualitative and quantitative evidence to further support the validity and interpretation of the 5-domain NPC Clinical Severity Scale, particularly the swallow domain.
CRL要求进一步提供定性和定量证据，以进一步支持5个领域NPC临床严重度评分的有效性和解释，尤其是吞咽领域。
Zevra Therapeutics resubmitted its New Drug Application (NDA) for arimoclomol to the FDA in December 2023.
Zevra Therapeutics在2023年12月向FDA重新提交了arimoclomol的新药申请(NDA)。
William Blair has increased the probability of success of arimoclomol in NPC to 90% from 80%, citing the positive AdCom vote.
William Blair将arimoclomol在NPC治疗中的成功可能性从80％上调至90％, 引用AdCom的正面投票结果。
The analyst highlights that Zevra is evolving into a fully integrated biotechnology company with a balanced portfolio of marketed products and late-stage development assets focused on rare diseases.
该分析师强调,Zevra正在成为一个拥有平衡组合罕见疾病市场产品和后期发展资产的全面综合型生物技术公司。
"We believe the launch of Olpruva in 2024 represents an attractive market opportunity where the company can see some early commercial success ahead of the potential approval of its second asset,arimoclomol, which is currently under FDA review for NPC," the analyst adds.
该分析师补充道:"我们相信2024年Olpruva的推出代表着一个有吸引力的市场机遇, 公司可以在NPC获批之前看到一些早期商业成功, 目前正在接受FDA审查的第二个资产arimoclomol。
In December 2022, the FDA approved Olpruva for urea cycle disorders.
2022年12月, FDA批准了Olpruva用于尿素循环紊乱。
2024 represents a significant year for Zevra as new management continues to guide the company through a transformational period with one launch underway, a potential approval and second launch later in the year, and updates from the clinical pipeline, William Blair notes and keeps the Outperform rating.
William Blair指出, 2024年将是Zevra的重要一年,新管理层继续引导该公司进行转型期, 已经有一个推出在进行中, 一项潜在的获批和第二个推出将在今年晚些时候进行, 同时, 临床流水线也有更新, 该机构保持表现优异评级。
Price Action: ZVRA stock is up 16.7% at $7.35 during the premarket session at last check Monday.
周一盘前交易时，ZVRA股票上涨16.7％，报7.35美元。
Photo via Shutterstock
图片来自shutterstock。
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