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将于8月2日召开与新成立的遗传代谢疾病咨询委员会（GeMDAC）的会议，审查Zevra Therapeutics Inc.（NASDAQ：ZVRA）的arimoclomol治疗Niemann-Pick病C型（NPC）的营销申请。
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的申请已被指定为处方药用户费法（PDUFA）行动日期，截止日期为9月21日，此前截止日期为6月21日。
Previously known as KemPharm, Zevra Therapeutics acquired arimoclomol from Orphazyme for $12.8 million.
以前被称为KemPharm，Zevra Therapeutics以1280万美元从Orphazyme收购了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。
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表示数据支持arimoclomol的疗效和安全性，并预计在支持批准的会议上会有重要的患者倡导活动。
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，与arimoclomol之前的申请不同，患者团体已经发出支持信，包括来自国家Niemann-Pick病基金会和其他六个NPC倡导和研究机构的请愿书，其中附有将近1000名NPC患者、看护者和医生的签名。
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.
如果获批，尽管NPC属于超稀有疾病，但arimoclomol将为Zevra带来重要的商业机会。该分析师补充称，据信约有900名美国患者受到影响，其中估计有300-400人目前已被诊断出来。
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.
公司在今年早些时候建立了Olpruva的商业基础设施。通过美国早期接触计划，约有70名患者正在进行治疗，该分析师预计将会有一个强劲的推出。
This will involve converting these patients to full-launch participants and attracting new patients, many of whom have already shown interest.
这将涉及将这些患者转化为全面推出的参与者，并吸引许多已经表现出兴趣的新患者。
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(纳斯达克股票代码:XOMA)少量的里程碑和中单数字的版税。鉴于患者群体的超级孤儿病性质，价格将是所有利益相关者的关键变量，待批准。
William Blair reiterates the Outperform rating.
William Blair重申Outperform评级。
Price Action: ZVRA shares are up 17.4% at $5.27 at last check Tuesday.
价格行动：截至周二最后一次核对时，ZVRA股价上涨了17.4%，为5.27美元。