EMA grants orphan drug designations to Alnylam’s ALN-AT3 for haemophilia treatment

7 August 2014 (Last Updated August 7th, 2014 18:30)

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.

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 5x15 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."

"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.