Ocugen's Retina Gene Therapy Drug Gets Rare Disease Designation From FDA

Dow Jones

May 27, 2025

By Adriano Marchese

Ocugen's drug to treat retina diseases received a rare pediatric disease designation from U.S. regulators.

The biotechnology said Tuesday that the Food and Drug Administration granted the designation to OCU410ST, a gene therapy for the treatment of ABCA4-associated retinopathies, or genetic retinal disorders.

Previously, OCU410ST received orphan drug designations for the treatment of these retinopathies from the FDA and European Medicines Agency.

With the new designation, Ocugen may be awarded a priority review voucher, if the program is reauthorized by the U.S. Congress, the company said, which is a system designed to encourage drug development for rare diseases.

Write to Adriano Marchese at adriano.marchese@wsj.com

(END) Dow Jones Newswires

May 27, 2025 08:18 ET (12:18 GMT)

At the request of the copyright holder, you need to log in to view this content

Disclaimer: Investing carries risk. This is not financial advice. The above content should not be regarded as an offer, recommendation, or solicitation on acquiring or disposing of any financial products, any associated discussions, comments, or posts by author or other users should not be considered as such either. It is solely for general information purpose only, which does not consider your own investment objectives, financial situations or needs. TTM assumes no responsibility or warranty for the accuracy and completeness of the information, investors should do their own research and may seek professional advice before investing.

Most Discussed

{"basename":"","ssrTDKData":{"titleTemplate":"%s - Tiger Brokers","title":"Tiger Brokers | Global Stocks, Options & Futures Trading App","description":"Tiger Brokers, one-stop investment in US stocks, SGX stocks, HK stocks, A-shares & other global assets. One of the best stock trading platforms in Singapore.","keywords":"tiger brokers,tiger trade,tiger brokers singapore,broker online,stock trading in singapore,share trading singapore,brokerage firm singapore,trading app,stock broker singapore,stock trading platforms,trading account","social":{"ogDescription":"Tiger Brokers, one-stop investment in US stocks, SGX stocks, HK stocks, A-shares & other global assets. One of the best stock trading platforms in Singapore.","ogImage":"https://c1.itigergrowtha.com/portal5/static/media/og-logo.be62fbe1.png","ogUrl":"https://www.itiger.com/news/2538110466"},"companyName":"Tiger Brokers"},"pageData":{"isMobile":false,"isTiger":false,"isTTM":true,"region":"SGP","license":"TBSG","edition":"fundamental"},"isCrawlerRequest":true,"__swrFallback__":{"@#url:\"https://stock-news.skytigris.cn/v3/news\",params:#id:\"2538110466\",edition:\"fundamental\",auth_exemption:1,,,undefined,":{"share":"https://ttm.financial/m/news/2538110466?lang=en_US&edition=fundamental","thumbnail":"","is_english":true,"pubTime":"2025-05-27 20:18","share_image_url":"https://static.laohu8.com/e9f99090a1c2ed51c021029395664489","id":"2538110466","market":"other","top_or_hot":-1,"title":"Ocugen's Retina Gene Therapy Drug Gets Rare Disease Designation From FDA","media":"Dow Jones","content":"<font class=\"NormalMinus1\" face=\"Arial\">\n<pre>\n \n</pre>\n<p>\n  By Adriano Marchese \n</p>\n<pre>\n \n</pre>\n<p>\n  <a href=\"https://laohu8.com/S/OCGN\">Ocugen</a>'s drug to treat retina diseases received a rare pediatric disease designation from U.S. regulators. \n</p>\n<p>\n  The biotechnology said Tuesday that the Food and Drug Administration granted the designation to OCU410ST, a gene therapy for the treatment of ABCA4-associated retinopathies, or genetic retinal disorders. \n</p>\n<p>\n  Previously, OCU410ST received orphan drug designations for the treatment of these retinopathies from the FDA and European Medicines Agency. \n</p>\n<p>\n  With the new designation, Ocugen may be awarded a priority review voucher, if the program is reauthorized by the U.S. Congress, the company said, which is a system designed to encourage drug development for rare diseases. \n</p>\n<pre>\n \n</pre>\n<p>\n  Write to Adriano Marchese at adriano.marchese@wsj.com \n</p>\n<pre>\n \n</pre>\n<p>\n  (END) Dow Jones Newswires\n</p>\n<p>\n  May 27, 2025 08:18 ET (12:18 GMT)\n</p>\n<p>\n  Copyright (c) 2025 Dow Jones &amp; Company, Inc.\n</p>\n</font>","source":null,"html":"<!DOCTYPE html>\n<html>\n<head>\n<meta http-equiv=\"Content-Type\" content=\"text/html; charset=utf-8\" />\n<meta name=\"viewport\" content=\"width=device-width,initial-scale=1.0,minimum-scale=1.0,maximum-scale=1.0,user-scalable=no\"/>\n<meta name=\"format-detection\" content=\"telephone=no,email=no,address=no\" />\n<title>Ocugen's Retina Gene Therapy Drug Gets Rare Disease Designation From FDA</title>\n<style type=\"text/css\">\na,abbr,acronym,address,applet,article,aside,audio,b,big,blockquote,body,canvas,caption,center,cite,code,dd,del,details,dfn,div,dl,dt,\nem,embed,fieldset,figcaption,figure,footer,form,h1,h2,h3,h4,h5,h6,header,hgroup,html,i,iframe,img,ins,kbd,label,legend,li,mark,menu,nav,\nobject,ol,output,p,pre,q,ruby,s,samp,section,small,span,strike,strong,sub,summary,sup,table,tbody,td,tfoot,th,thead,time,tr,tt,u,ul,var,video{ font:inherit;margin:0;padding:0;vertical-align:baseline;border:0 }\nbody{ font-size:16px; line-height:1.5; color:#999; background:transparent; }\n.wrapper{ overflow:hidden;word-break:break-all;padding:10px; }\nh1,h2{ font-weight:normal; line-height:1.35; margin-bottom:.6em; }\nh3,h4,h5,h6{ line-height:1.35; margin-bottom:1em; }\nh1{ font-size:24px; }\nh2{ font-size:20px; }\nh3{ font-size:18px; }\nh4{ font-size:16px; }\nh5{ font-size:14px; }\nh6{ font-size:12px; }\np,ul,ol,blockquote,dl,table{ margin:1.2em 0; }\nul,ol{ margin-left:2em; }\nul{ list-style:disc; }\nol{ list-style:decimal; }\nli,li p{ margin:10px 0;}\nimg{ max-width:100%;display:block;margin:0 auto 1em; }\nblockquote{ color:#B5B2B1; border-left:3px solid #aaa; padding:1em; }\nstrong,b{font-weight:bold;}\nem,i{font-style:italic;}\ntable{ width:100%;border-collapse:collapse;border-spacing:1px;margin:1em 0;font-size:.9em; }\nth,td{ padding:5px;text-align:left;border:1px solid #aaa; }\nth{ font-weight:bold;background:#5d5d5d; }\n.symbol-link{font-weight:bold;}\n/* header{ border-bottom:1px solid #494756; } */\n.title{ margin:0 0 8px;line-height:1.3;color:#ddd; }\n.meta {color:#5e5c6d;font-size:13px;margin:0 0 .5em; }\na{text-decoration:none; color:#2a4b87;}\n.meta .head { display: inline-block; overflow: hidden}\n.head .h-thumb { width: 30px; height: 30px; margin: 0; padding: 0; border-radius: 50%; float: left;}\n.head .h-content { margin: 0; padding: 0 0 0 9px; float: left;}\n.head .h-name {font-size: 13px; color: #eee; margin: 0;}\n.head .h-time {font-size: 11px; color: #7E829C; margin: 0;line-height: 11px;}\n.small {font-size: 12.5px; display: inline-block; transform: scale(0.9); -webkit-transform: scale(0.9); transform-origin: left; -webkit-transform-origin: left;}\n.smaller {font-size: 12.5px; display: inline-block; transform: scale(0.8); -webkit-transform: scale(0.8); transform-origin: left; -webkit-transform-origin: left;}\n.bt-text {font-size: 12px;margin: 1.5em 0 0 0}\n.bt-text p {margin: 0}\n</style>\n</head>\n<body>\n<div class=\"wrapper\">\n<header>\n<h2 class=\"title\">\nOcugen's Retina Gene Therapy Drug Gets Rare Disease Designation From FDA\n</h2>\n\n<h4 class=\"meta\">\n\n\n<div class=\"head\" \">\n\n\n<div class=\"h-thumb\" style=\"background-image:url(https://static.tigerbbs.com/150f88aa4d182df19190059f4a365e99);background-size:cover;\"></div>\n\n<div class=\"h-content\">\n<p class=\"h-name\">Dow Jones </p>\n<p class=\"h-time\">2025-05-27 20:18</p>\n</div>\n\n</div>\n\n\n</h4>\n\n</header>\n<article>\n<font class=\"NormalMinus1\" face=\"Arial\">\n<pre>\n \n</pre>\n<p>\n  By Adriano Marchese \n</p>\n<pre>\n \n</pre>\n<p>\n  <a href=\"https://laohu8.com/S/OCGN\">Ocugen</a>'s drug to treat retina diseases received a rare pediatric disease designation from U.S. regulators. \n</p>\n<p>\n  The biotechnology said Tuesday that the Food and Drug Administration granted the designation to OCU410ST, a gene therapy for the treatment of ABCA4-associated retinopathies, or genetic retinal disorders. \n</p>\n<p>\n  Previously, OCU410ST received orphan drug designations for the treatment of these retinopathies from the FDA and European Medicines Agency. \n</p>\n<p>\n  With the new designation, Ocugen may be awarded a priority review voucher, if the program is reauthorized by the U.S. Congress, the company said, which is a system designed to encourage drug development for rare diseases. \n</p>\n<pre>\n \n</pre>\n<p>\n  Write to Adriano Marchese at adriano.marchese@wsj.com \n</p>\n<pre>\n \n</pre>\n<p>\n  (END) Dow Jones Newswires\n</p>\n<p>\n  May 27, 2025 08:18 ET (12:18 GMT)\n</p>\n<p>\n  Copyright (c) 2025 Dow Jones &amp; Company, Inc.\n</p>\n</font>\n\n</article>\n</div>\n</body>\n</html>\n","isBrief":false,"type":0,"news_type":1,"symbol":"OCGN","symbol_name":"Ocugen","start_time":0,"source_url":"https://dowjonesnews.com/newdjn/logon.aspx?AL=N","article_id":"2538110466","we_media_id":"106","thumbnails":[],"rights":null,"url":"https://stock-news.laohu8.com/highlight/detail?id=2538110466","pubTimestamp":1748348280,"columns":[{"name":"Policy And Regulatory","id":32,"label_key":"policyRegulatory"}],"sourceInfo":null,"weMediaInfo":{"media_name":"Dow Jones","introduction":"Dow Jones publishes the world’s most trusted business news and financial information in a variety of media.","home_visible":0,"id":"106","head_image":"https://static.tigerbbs.com/150f88aa4d182df19190059f4a365e99"},"summary":"Ocugen's drug to treat retina diseases received a rare pediatric disease designation from U.S. regulators.The biotechnology said Tuesday that the Food and Drug Administration granted the designation to OCU410ST, a gene therapy for the treatment of ABCA4-associated retinopathies, or genetic retinal disorders.Previously, OCU410ST received orphan drug designations for the treatment of these retinopathies from the FDA and European Medicines Agency.With the new designation, Ocugen may be awarded a priority review voucher, if the program is reauthorized by the U.S. Congress, the company said, which is a system designed to encourage drug development for rare diseases.","collect":0,"end_time":0,"defaultTopTitle":"","property":[],"viewcount":null,"language":"en","relate_stocks":{"OCGN":"Ocugen","BK4568":"美国抗疫概念","BK4563":"昨日强势股","BK4139":"生物科技"},"translate_title":"Ocugen视网膜基因治疗药物获得FDA罕见病认定","themeId":null,"isJumpTheme":false,"ttsUrl":null,"symbols_score_info":{"OCGN":1},"content_text":"By Adriano Marchese \n\n\n \n\n\nOcugen's drug to treat retina diseases received a rare pediatric disease designation from U.S. regulators. \n\n\n  The biotechnology said Tuesday that the Food and Drug Administration granted the designation to OCU410ST, a gene therapy for the treatment of ABCA4-associated retinopathies, or genetic retinal disorders. \n\n\n  Previously, OCU410ST received orphan drug designations for the treatment of these retinopathies from the FDA and European Medicines Agency. \n\n\n  With the new designation, Ocugen may be awarded a priority review voucher, if the program is reauthorized by the U.S. Congress, the company said, which is a system designed to encourage drug development for rare diseases. \n\n\n \n\n\n  Write to Adriano Marchese at adriano.marchese@wsj.com \n\n\n \n\n\n  (END) Dow Jones Newswires\n\n\n  May 27, 2025 08:18 ET (12:18 GMT)\n\n\n  Copyright (c) 2025 Dow Jones & Company, Inc.","kind":"highlight","is_publish_news":true,"is_publish_highlight":false,"is_publish_live":false,"is_publish_wemedia":null,"editions":null,"column":"","sentiment":"1","news_tag":"policyRegulatory","news_rank":0,"symbols":[],"gpt_button":0,"need_auth":true,"code":"91000000","status":"200"}}}