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全球领先的RNAi药物开发公司——Alnylam制药8月20日宣布，FDA已授予ALN-AT3孤儿药地位（Orphan Drug Designation，ODD），用于治疗A型血友病患者。上周，FDA也已授予ALN-AT3治疗B型血友病的孤儿药地位。
目前，Alnylam正开发ALN-AT3作为一种皮下注射RNAi疗法，靶向抗凝血酶（AT），用于治疗血友病：包括A型血友病、B型血友病、伴有抑制剂的A或B型血友病、及其他罕见出血性疾病（RBD）。
ALN-AT3采用了Alnylam制药专有的半乳糖胺共轭递送平台，能够通过皮下注射给药。该公司计划在2013年第四季度提交研究性新药申请（IND），并于2014年初启动I期临床试验。
临床前数据表明，ALN-AT3能够使凝血酶的生成正常化，并能够改善血友病小鼠的止血，同时还可以完全纠正一种非人类灵长类动物（NHP）血友病"抑制剂"模型中凝血酶的生成。
关于Alnylam制药
Alnylam是一家生物制药公司，开发基于RNA干扰（RNAi）的新型疗法。该公司在研的其他药物还包括：ALN-TTR，开发用于治疗甲状腺素介导的淀粉样变性（ATTR）；ALN-PCS，开发用于治疗严重高胆固醇血症；AlN-HPN，开发用于治疗难治性贫血的治疗。
英文原文：Alnylam Receives Additional Orphan Drug Designation from U.S. Food & Drug Administration for ALN-AT3，an RNAi Therapeutic for the Treatment of Hemophilia
Orphan Drug Designation for ALN-AT3 Now Includes Treatment of Both Hemophilia A and Hemophilia B –
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Aug. 20, 2013-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that the U.S. Food & Drug Administration (FDA) has granted an Orphan Drug Designation (ODD) to ALN-AT3 as a therapeutic for the treatment of hemophilia A. As reported last week, the FDA has also granted ODD to ALN-AT3 for the treatment of hemophilia B. Alnylam is developing ALN-AT3, a subcutaneously administered RNAi therapeutic targeting antithrombin (AT), for the treatment of hemophilia – including hemophilia A, hemophilia B, and hemophilia A or B with “inhibitors” – and other Rare Bleeding Disorders (RBD).
“We are very pleased that the FDA has granted Orphan Drug Designation for ALN-AT3 now for both the treatment of hemophilia A and hemophilia B. As a subcutaneously delivered RNAi therapeutic, we believe it represents an innovative approach for the management of hemophilia and has great potential to make a meaningful impact in the treatment of this often debilitating bleeding disorder,” said Saraswathy (Sara) Nochur, Ph.D., Senior Vice President, Regulatory Affairs and Quality Assurance at Alnylam. “ALN-AT3 is a key program in our ‘Alnylam 5x15’ product development and commercialization strategy, and we look forward to advancing this promising RNAi therapeutic into the clinic in the months to come.”
At the recent Congress of the International Society on Thrombosis and Haemostasis, Alnylam presented pre-clinical data demonstrating that ALN-AT3 can normalize thrombin generation and improve hemostasis in hemophilia mice and can fully correct thrombin generation in a non-human primate (NHP) hemophilia “inhibitor” model. ALN-AT3 utilizes the company’s proprietary GalNAc conjugate delivery platform, enabling subcutaneous dose administration. Alnylam plans to file an investigational new drug (IND) application for ALN-AT3 in the fourth quarter of 2013 and initiate a Phase I clinical trial in early 2014.
The FDA Office of Orphan Products Development (OOPD) mission is to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. OOPD provides incentives for sponsors to develop products for rare diseases. The Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S.