Biopharmaceutical company Alnylam Pharmaceuticals has received orphan drug designations for ALN-AT3 from the European Medicines Agency (EMA) Committee to treat haemophilia A and B.
ALN-AT3 is a subcutaneously administered RNAi therapeutic targeting antithrombin (AT) for treating haemophilia and other rare bleeding disorders (RBD).
Alnylam Pharmaceuticals regulatory affairs and quality assurance senior vice-president Saraswathy Nochur said: "We are very pleased to have received orphan drug designations from the EMA COMP for ALN-AT3, a key programme in our Alnylam 5×15 product development and commercialisation strategy.
"We believe that our subcutaneously delivered RNAi therapeutic represents an innovative approach for the management of haemophilia and has great potential to make a meaningful impact in the treatment of this often debilitating bleeding disorder."
Alnylam noted that ALN-AT3 is currently being investigated in a multinational Phase I trial.
The company presented positive top-line data from part A of the study performed in healthy adult volunteers at the World Federation of Haemophilia 2014 World Congress held in May.
A total of 18 people with moderate to severe haemophilia A or B have been enrolled in part B open-label, multi-dose, dose-escalation study.
This study’s primary objective is to evaluate the safety and tolerability of three weekly doses of subcutaneously administered ALN-AT3 in haemophilia subjects.
The clinical activity assessment, determined by the knockdown of circulating AT levels and increase in thrombin generation at pharmacologic doses of ALN-AT3, makes up the secondary objective.
Results from part B of the study are expected to be presented by the end of the year.