奥库根公司的OCU410ST获得ABCA4相关视网膜病变的罕见儿科疾病认定Ocugen’s OCU410ST Granted Rare Pediatric Disease Designation for ABCA4-Associated Retinopathies

动脉网2025-05-28

Ocugen’s OCU410ST Granted Rare Pediatric Disease Designation for ABCA4-Associated Retinopathies奥库根公司的OCU410ST获得ABCA4相关视网膜病的罕见儿科疾病认定May 28, 20252025年5月28日The U.S. Food and Drug Administration (FDA) has...

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These include Stargardt disease, retinitis pigmentosa 19, and cone-rod dystrophy 3.美国食品药品监督管理局已授予Ocugen公司的OCU410ST罕见儿科疾病认定，用于治疗ABCA4相关视网膜病变。这些认可强调了该疗法在应对目前尚无FDA批准治疗方案的罕见视网膜疾病方面的潜力。","collect":0,"end_time":0,"defaultTopTitle":"qq.com","property":[],"viewcount":null,"language":"zh","relate_stocks":{"BK4568":"美国抗疫概念","BK4563":"昨日强势股","ASB":"Associated Banc-Corp","BK4139":"生物科技","OCGN":"Ocugen","BK4211":"区域性银行"},"translate_title":"Ocugen's OCU410ST obtains Rare Pediatric Disease Designation for ABCA4-Associated Retinopathy Ocugen's OCU410ST Granted Rare Pediatric Disease Designation for ABCA4-Associated Retinopathy","themeId":null,"isJumpTheme":false,"ttsUrl":null,"symbols_score_info":{"OCGN":1,"ASB":0.9},"content_text":"Ocugen’s OCU410ST Granted Rare Pediatric Disease Designation for ABCA4-Associated Retinopathies奥库根公司的OCU410ST获得ABCA4相关视网膜病的罕见儿科疾病认定May 28, 20252025年5月28日The U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to Ocugen’s OCU410ST for the treatment of ABCA4-associated retinopathies. These include Stargardt disease, retinitis pigmentosa 19, and cone-rod dystrophy 3.美国食品药品监督管理局（FDA）已授予Ocugen公司的OCU410ST罕见儿科疾病认定（RPDD），用于治疗ABCA4相关视网膜病变。这些疾病包括斯特格病、视网膜色素变性19型和锥杆细胞营养不良3型。Building on Previous Regulatory Milestones建立在以往监管里程碑的基础上OCU410ST had previously received Orphan Drug Designation from both the FDA and the European Medicines Agency (EMA) for the treatment of ABCA4-associated retinopathies. These recognitions emphasize the therapy’s potential in addressing rare retinal diseases that currently lack FDA-approved treatments..OCU410ST此前已获得美国食品药品监督管理局（FDA）和欧洲药品管理局（EMA）授予的孤儿药资格，用于治疗ABCA4相关视网膜病变。这些认可强调了该疗法在应对目前尚无FDA批准治疗方案的罕见视网膜疾病方面的潜力。Mechanism of Action: AAV-Mediated RORA Gene Delivery作用机制：AAV介导的RORA基因递送OCU410ST is based on an adeno-associated virus (AAV) delivery platform specifically designed for retinal gene therapy. It delivers the RORA (RAR-Related Orphan Receptor A) gene, which regulates key pathways involved in Stargardt disease, such as:OCU410ST基于一种专门设计用于视网膜基因治疗的腺相关病毒（AAV）递送平台。它递送RORA（RAR相关孤儿受体A）基因，该基因调控与斯塔加特病相关的关键通路，例如：• Lipofuscin formation• 脂褐素形成• Oxidative stress• 氧化应激• Complement activation• 补体激活• Inflammation• 炎症• Cell survival mechanisms• 细胞存活机制Company Leadership Emphasizes Urgency for Treatment公司领导强调治疗的紧迫性Shankar Musunuri, PhD, MBA, Chairman, CEO, and Co-Founder of Ocugen, commented in a press release:奥库根公司董事长、首席执行官兼联合创始人尚卡尔·穆苏努里博士、工商管理硕士在新闻稿中评论道：“This latest designation for OCU410ST reaffirms the urgency of providing a therapeutic option to Stargardt patients who have no FDA-approved treatment available.”“OCU410ST的这一最新认定再次确认了为斯塔加特病患者提供治疗选择的紧迫性，因为目前尚无FDA批准的治疗方法。”Implications of the Rare Pediatric Disease Designation罕见儿科疾病认定的含义With this designation, Ocugen becomes eligible for a Priority Review Voucher (PRV) — if the PRV program is reauthorized by the U.S. Congress. A PRV can either be used to accelerate FDA review for another product or sold to another pharmaceutical company. Historically, these vouchers sell for $100 million or more..有了这一指定，Ocugen 将有资格获得优先审查券（PRV）——如果美国国会重新授权 PRV 计划。PRV 可以用于加速 FDA 对另一产品的审查，也可以出售给其他制药公司。历史上，这些券的售价通常在 1 亿美元或以上。Next Steps in Clinical Development临床开发的下一步Ocugen plans to initiate a Phase 2/3 pivotal confirmatory trial in the coming weeks. The company also aims to submit a Biologics License Application (BLA) for OCU410ST in 2027, marking a significant step toward bringing the therapy to market.奥库根计划在未来几周内启动一项二期/三期关键确证性试验。该公司还计划在2027年为OCU410ST提交生物制品许可申请（BLA），这标志着将该疗法推向市场的重要一步。","kind":"news","is_publish_news":true,"is_publish_highlight":false,"is_publish_live":false,"is_publish_wemedia":null,"editions":null,"column":"","sentiment":"1","news_tag":"","news_rank":0,"isVideo":false,"video":null,"symbols":[],"gpt_button":1,"need_auth":false,"need_login_tip":false,"code":"91000000","status":"200"},"commentList":[],"isCommentEnd":true,"newsSizeData":{"likeSize":0,"commentSize":0,"repostSize":0,"favoriteSize":0,"likeStatus":false,"favoriteStatus":false},"APP":{"userAgent":"Mozilla/5.0 AppleWebKit/537.36 (KHTML, like Gecko; compatible; ClaudeBot/1.0; +claudebot@anthropic.com)","isDev":false,"isTTM":false,"tenantId":"TBCN","deviceId":"web-server-community-laohu8-v3","version":"4.43.1","shortVersion":"4.43.1","platform":"web","vendor":"web","appName":"laohu8","isIOS":false,"isAndroid":false,"isTiger":false,"isTHS":false,"isWeiXin":false,"isWeiXinMini":false,"isWeiBo":false,"isQQ":false,"isBaiduSwan":false,"isBaiduBox":false,"isDingTalk":false,"isToutiao":false,"isOnePlus":false,"isHuaWei":false,"isXiaomi":false,"isXiaomiWebView":false,"isOppo":false,"isVivo":false,"isSamsung":false,"isMobile":false},"href":"/m/news/2538476900","isCrawlerRequest":true}