FDA Convenes Expert Panel Meeting For Zevra Therapeutics' Arimoclomol, Analyst Optimistic About Approval Despite Small Patient Base
FDA Convenes Expert Panel Meeting For Zevra Therapeutics' Arimoclomol, Analyst Optimistic About Approval Despite Small Patient Base
The FDA will convene a meeting with the recently formed Genetic Metabolic Diseases Advisory Committee (GeMDAC) on August 2 to review the marketing application for Zevra Therapeutics Inc.'s (NASDAQ:ZVRA) arimoclomol for Niemann-Pick disease type C (NPC).
FDA將於8月2日召開與新成立的遺傳代謝疾病諮詢委員會（GeMDAC）的會議，審查Zevra Therapeutics Inc.（NASDAQ：ZVRA）的arimoclomol治療Niemann-Pick病C型（NPC）的營銷申請。
The application for arimoclomol has been assigned a Prescription Drug User Fee Act (PDUFA) action date of September 21, which was extended from June 21.
arimoclomol的申請已被指定爲處方藥用戶費法（PDUFA）行動日期，截止日期爲9月21日，此前截止日期爲6月21日。
Previously known as KemPharm, Zevra Therapeutics acquired arimoclomol from Orphazyme for $12.8 million.
以前被稱爲KemPharm，Zevra Therapeutics以1280萬美元從Orphazyme收購了arimoclomol。
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。
William Blair says the data supports the efficacy and safety of arimoclomol supports approval and expects significant patient advocacy at the meeting in support of approval.
William Blair表示數據支持arimoclomol的療效和安全性，並預計在支持批准的會議上會有重要的患者倡導活動。
Additionally, unlike the prior filing for arimoclomol, patient groups have already sent letters of support, including a petition from the National Niemann-Pick Disease Foundation and six other NPC advocacy and research organizations with nearly 1,000 signatures from NPC patients, caregivers, and physicians.
此外，與arimoclomol之前的申請不同，患者團體已經發出支持信，包括來自國家Niemann-Pick病基金會和其他六個NPC倡導和研究機構的請願書，其中附有將近1000名NPC患者、看護者和醫生的簽名。
If approved, arimoclomol would represent a significant commercial opportunity for Zevra despite the ultra-rare nature of NPC. The analyst adds that NPC is believed to affect around 900 patients in the U.S., of whom an estimated 300-400 are currently diagnosed.
如果獲批，儘管NPC屬於超稀有疾病，但arimoclomol將爲Zevra帶來重要的商業機會。該分析師補充稱，據信約有900名美國患者受到影響，其中估計有300-400人目前已被診斷出來。
The company established a commercial infrastructure for launching Olpruva earlier this year. With about 70 patients on therapy through the early access program in the U.S., the analyst expects a strong launch.
公司在今年早些時候建立了Olpruva的商業基礎設施。通過美國早期接觸計劃，約有70名患者正在進行治療，該分析師預計將會有一個強勁的推出。
This will involve converting these patients to full-launch participants and attracting new patients, many of whom have already shown interest.
這將涉及將這些患者轉化爲全面推出的參與者，並吸引許多已經表現出興趣的新患者。
Zevra owes modest milestones and mid-single-digit royalties to XOMA Corporation (NASDAQ:XOMA). Given the ultra-orphan nature of the patient population, pricing will be a key variable for all stakeholders involved, pending approval.
Zevra欠XOMA Corporation(納斯達克股票代碼:XOMA)少量的里程碑和中單數字的版稅。鑑於患者群體的超級孤兒病性質，價格將是所有利益相關者的關鍵變量，待批准。
William Blair reiterates the Outperform rating.
William Blair重申Outperform評級。
Price Action: ZVRA shares are up 17.4% at $5.27 at last check Tuesday.
價格行動：截至週二最後一次覈對時，ZVRA股價上漲了17.4%，爲5.27美元。