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的基因代謝病顧問委員會(GeMDAC)在星期五投票通過了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.
委員會投票贊成(11票贊成, 5票反對), 認爲數據支持arimoclomol有效治療尼曼-皮克病C型(NPC)患者, 這是一種罕見的進行性遺傳性疾病, 其特點是身體無法將膽固醇和其他脂質物質運輸到細胞內。
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將在完成對arimoclomol NDA的獨立審查時考慮委員會的建議, 但GeMDAC的反饋不對該機構具有約束力。
The arimoclomol NDA has been assigned a September 21 Prescription Drug User Fee Act (PDUFA) action date.
arimoclomol NDA已被指定爲9月21日處方藥用戶費用法案(PDUFA)的行動日期。
In 2021, the FDA issued a complete response letter to arimoclomol as a treatment option for Niemann-Pick disease type C.
2021年，FDA發出完整的答覆信，稱arimoclomol不是治療Niemann-Pick病C型的選擇。
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要求進一步提供定性和定量證據，以進一步支持5個領域NPC臨床嚴重度評分的有效性和解釋，尤其是吞嚥領域。
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的新藥申請(NDA)。
William Blair has increased the probability of success of arimoclomol in NPC to 90% from 80%, citing the positive AdCom vote.
William Blair將arimoclomol在NPC治療中的成功可能性從80％上調至90％, 引用AdCom的正面投票結果。
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.
該分析師強調,Zevra正在成爲一個擁有平衡組合罕見疾病市場產品和後期發展資產的全面綜合型生物技術公司。
"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.
該分析師補充道:"我們相信2024年Olpruva的推出代表着一個有吸引力的市場機遇, 公司可以在NPC獲批之前看到一些早期商業成功, 目前正在接受FDA審查的第二個資產arimoclomol。
In December 2022, the FDA approved Olpruva for urea cycle disorders.
2022年12月, FDA批准了Olpruva用於尿素循環紊亂。
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年將是Zevra的重要一年,新管理層繼續引導該公司進行轉型期, 已經有一個推出在進行中, 一項潛在的獲批和第二個推出將在今年晚些時候進行, 同時, 臨床流水線也有更新, 該機構保持表現優異評級。
Price Action: ZVRA stock is up 16.7% at $7.35 during the premarket session at last check Monday.
週一盤前交易時，ZVRA股票上漲16.7％，報7.35美元。
Photo via Shutterstock
圖片來自shutterstock。
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