2024年8月22日，中美瑞康 (Ractigen Therapeutics) 宣布其小激活RNA (saRNA) 药物RAG-18获得美国食品药品监督管理局 (FDA) 的孤儿药认证 (Orphan Drug Designation, ODD) 。继上月RAG-18获得FDA儿科罕见病药物资格认定 (Rare Pediatric Disease Designation, RPDD) 之后，这一最新认证进一步确立了RAG-18创新性治疗杜氏肌营养不良 (Duchenne Muscular Dystrophy, DMD) 和贝氏肌营养不良 (Becker Muscular Dystrophy, BMD) 的潜力，无论DMD基因突变类型如何，RAG-18都可能成为一种具有可转化前景的治疗策略。这是全球首个获得FDA孤儿药认定的saRNA药物。
孤儿药认定由FDA授予，旨在鼓励用于治疗罕见病（在美国影响少于20万人的疾病）的药物和生物制品的开发。获得孤儿药认定的公司将享有多项开发激励措施，包括自市场批准之日起的七年市场独占权以及新药申请费用的豁免。
中美瑞康创始人兼首席执行官李龙承博士表示：“获得FDA的孤儿药认定是RAG-18达成的又一个关键里程碑。继获得儿科罕见病药物资格认定之后，这次认证进一步肯定了我们在RNA激活领域的开创性工作，也增强了我们为罕见病患者带来改变的决心。这一认定为我们注入了新的动力，我们将继续加速RAG-18的开发进程，致力于为全球DMD和BMD患者带来革命性的治疗选择。”
关于RAG-18
RAG-18是一款首创作用机制的双链saRNA药物，通过RNA激活（RNAa）机制特异性靶向激活肌肉细胞中UTRN基因表达。由UTRN基因编码的肌营养不良蛋白（Utrophin）在结构和功能上与抗肌萎缩蛋白（Dystrophin）相似，它的上调可以功能性替代 DMD 肌肉细胞中缺失的抗肌萎缩蛋白，从而治疗所有突变类型的DMD和BMD患者。临床前研究表明，采用中美瑞康自主开发的小核酸递送系统LiCO™并通过皮下注射，RAG-18能够显著减轻DMD模型小鼠的肌肉损伤，显示出在治疗DMD患者方面的潜力。今年7月，RAG-18已获美国FDA的儿科罕见病药物资格认定。
关于DMD
杜氏肌营养不良症（DMD）是一种严重的X染色体隐性遗传病，由编码抗肌蛋白的DMD基因突变引起。这种疾病主要影响男性儿童，患者通常在3至5岁时首次显示出肌肉炎症、纤维化和运动能力下降等症状。如果不进行治疗，多数患者将在20岁之前失去独立行走能力，并可能在30岁之前因心肌和膈肌（呼吸肌）衰竭而死亡。DMD的全球发病率约为每3600至6000名新生男婴中有一例，中国的患者数量约为70,000人。目前针对DMD的治疗方法包括反义寡核苷酸（ASO）介导的外显子跳跃、基因治疗和基因编辑，但这些疗法的效果非常有限，因此在DMD药物开发方面仍存在巨大的临床需求。
关于RNA激活
RNA激活（RNAa）是中美瑞康创始人李龙承博士及其团队在国际上开创，并已经过临床验证的平台技术。该技术利用靶向基因启动子区域的双链RNA来激活基因表达，以恢复治疗性蛋白的水平。RNA激活技术是制药领域十分稀缺的平台技术，在药物开发上具有广阔的应用前景，包括遗传性疾病、慢性疾病、代谢性疾病、心脑血管疾病、肿瘤等。
中美瑞康
Ractigen Therapeutics
中美瑞康（Ractigen Therapeutics）是一家立足于中国和面向全球市场的平台型新药研发公司，致力于开发突破性小核酸药物与疾病治疗方法。中美瑞康是全球少数同时掌握有肝内与肝外递送的小核酸药企之一，开发出了具有独立自主知识产权的SCAD™、LiCO™等多个具有国际领先水平的小核酸药物递送平台技术。基于RNA激活技术和自主开发的Smart-TTC saRNA药物开发平台，公司建立了具有高度差异化的小核酸药物管线，适应症涵盖神经退行性与神经肌肉疾病、肿瘤、代谢与血液系统疾病等，为诸多疾病领域中无法成药的靶点、无法治愈的疾病提供创新型治疗方案。详情请访问官网 www.ractigen.com。
Ractigen Therapeutics Announces U.S. FDA Orphan Drug Designation (ODD) granted to RAG-18 for the treatment of Duchenne Muscular Dystrophy and Becker Muscular Dystrophy
JIANGSU, China, August 22, 2024 – Ractigen Therapeutics, a pioneering developer of small activating RNA (saRNA) therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to RAG-18, one of the company’s lead saRNA product candidates. This designation follows the recent Rare Pediatric Disease Designation (RPDD) granted to RAG-18 last month, further solidifying the potential of this novel therapeutic strategy for Duchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy (BMD) caused by any mutation of the DMD gene. These milestones establish RAG-18 as the world’s first saRNA drug to achieve such significant regulatory recognition from the FDA.
Orphan drug designation is granted by the FDA to a drug or biologic intended to treat a rare disease or condition, which generally includes a disease or condition that affects fewer than 200,000 individuals in the U.S. The designation provides companies with development incentives, including a seven-year marketing exclusivity from the date of market approval and a waiver of the New Drug Application fee.
Dr. Long-Cheng Li, Founder and CEO of Ractigen Therapeutics, stated: “Receiving FDA Orphan Drug Designation marks a pivotal achievement for RAG-18. Combined with the recent Rare Pediatric Disease Designation, it reflects the groundbreaking work we’re doing with RNA activation (RNAa) and reinforces our commitment to making a real difference in the lives of those affected by rare diseases. This recognition fuels our determination to push forward with RAG-18’s development, aiming to bring innovative and life-changing treatments to DMD and BMD patients around the world.”
About RAG-18
RAG-18 is a first of its kind saRNA candidate designed to specifically target and activate UTRN gene expression in muscle cells via RNAa mechanism. The utrophin protein encoded by the UTRN gene is structurally and functionally similar to dystrophin, and its upregulation could potentially serve as a functional replacement for the missing dystrophin in DMD muscle cells, providing treatment for all DMD patients regardless of the specific mutation location. Preclinical data indicate that RAG-18, delivered through subcutaneous injection utilizing Ractigen’s proprietary LiCO™ (lipid-conjugated oligonucleotide) technology, has effectively mitigated muscle damage, demonstrating significant potential in treating DMD patients. RAG-18 has recently received RPDD from the U.S. FDA in July this year.
About DMD and BMD
Duchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy (BMD) are severe genetic disorders caused by mutations in the dystrophin gene, leading to the absence or insufficiency of functional dystrophin protein. This protein is essential for muscle fiber stability. Without it, muscle cells are easily damaged and cannot repair themselves, resulting in progressive muscle weakness and degeneration. The dystrophin gene, the largest in the human body, contains 79 exons. Current disease-modifying therapeutic approaches for DMD include antisense oligonucleotides (ASO) mediated exon skipping, gene therapy, and gene editing, with exon skipping being the most common strategy. However, these treatments have significant limitations, highlighting the critical need for innovative therapies that target the root cause of DMD to provide more effective and long-lasting benefits for patients.
About RNAa
RNAa is a clinically validated platform technology developed by Dr. Long-Cheng Li and his team. It utilizes saRNAs to target gene regulatory domains, activating gene expression and restoring therapeutic protein levels. This innovative technology holds vast potential for developing therapeutic drugs across various diseases, particularly where traditional methods fall short, including genetic disorders.
About Ractigen Therapeutics
A leader in saRNA drug development, Ractigen Therapeutics is at the forefront of developing saRNA drugs utilizing the RNAa mechanism to up-regulate endogenous gene expression. This innovative approach involves saRNA targeting specific genes to enhance transcription, thereby restoring normal protein functions. Ractigen's cutting-edge technology is pivotal in treating diseases unaddressable by conventional methods, such as those resulting from epigenetic silencing or gene downregulation. For more information, please visit our website at www.ractigen.com.
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