Ruijian Pharmaceutical's ophthalmology products are recognized as orphan drugs by the FDA, lighting up hope for rare diseases
On March 20, 2024, NouvSight001, the breakthrough ophthalmic product of Wuhan Ruijian Pharmaceutical Technology Co., Ltd.(Ruijian Pharmaceutical), was awarded Orphan Drug Designation (ODD) by the US FDA. 该产品面向“视网膜色素变性系列适应症”治疗，是睿健医药团队依托公司独特的“AI+化学诱导”平台，开发的首款诱导多能干细胞（iPSC）来源的眼科通用型细胞治疗产品。
NouvSight001所针对的视网膜色素变性（Retinitis Pigmentosa，RP），是一种可导致失明的遗传性眼科疾病。患病初期主要表现为夜盲，周边视野缺损等症状。当视野缺损逐渐恶化，将进一步导致管视角（视野狭隘），甚至完全失明。整个过程不可逆，临床上也尚无治愈该疾病的有效方法。
现有的研究结果显示，视网膜色素变性可由相关超过50种基因的任一变异引起，发病机制主要归于眼底视杆细胞进行性缺失及视锥细胞缺失，发病率约为1/4000，是世界卫生组织（WTO）定义的罕见病种。但 Even at an incidence rate of 1 in 4,000, more than 1.5 million patients worldwide suffer from increasing blindness. This is also the reason why NouvSight001 developed by Ruijian Pharmaceutical has attracted the attention of the FDA. 公开信息显示， 孤儿药认定（Orphan Drug Designation, ODD）是FDA孤儿药产品开发办公室（Office of Orphan Products Development，OOPD) 对符合条件的用于预防、治疗及诊断罕见病的药物（包括生物制品）授予的一种资格认定。获得孤儿药认定的药物在药物获批上市后享有7年市场独占权（marketing exclusivity），在临床研究阶段费用享受税收减免，并同时免除NDA/BLA申请费（PDUFA）。
The ophthalmology product NouvSight001 was awarded orphan drug designation by the US FDA, which is also a landmark milestone in the development of Ruijian Pharmaceutical.
