The European Medicines Agency (EMA) has granted orphan designation for Amsterdam Molecular Therapeutics' (AMT), a leader in the field of human gene therapy, gene therapy programme for the treatment of haemophilia B.

Orphan designation in the European Union provides several benefits including 10 years of market exclusivity from product launch and access to the central authorization procedure.

AMT’s haemophilia B programme, which consists of an adeno-associated viral (AAV) vector containing the human factor IX gene, is being investigated in a Phase I/II study conducted by St. Jude’s Children’s Research Hospital (Memphis, USA) and University College London (UK). Promising data from an initial 6 patients shows that gene therapy administration resulted in a reduced need for protein replacement treatment, the standard care for haemophilia patients. AMT is preparing for additional clinical development work to establish safety, tolerability and proof-of-concept with a factor IX gene therapy produced using its proprietary AAV production system.

“Orphan designation is an important milestone for our haemophilia B gene therapy programme and will provide additional support to our negotiations as we seek potential licensing partners,” said Jörn Aldag, CEO of AMT. “A successful gene therapy for haemophilia could dramatically change not only the lives of patients but also the current haemophilia market that is dominated by protein replacement therapies.”

AMT is a world leader in the development of human gene based therapies. The company has a product pipeline of gene therapy products in development for haemophilia B, acute intermittent porphyria, Parkinson’s disease and SanfilippoB.