Published on August 17, 2013 at 6:45 AM
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.
Source: Alnylam Pharmaceuticals, Inc.