Amsterdam Molecular Therapeutics Receives Orphan Designation in the European Union for Hemophilia B Gene Therapy‏

Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today that the European Medicines Agency (EMA) has granted orphan designation for its gene therapy program for the treatment of hemophilia 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 hemophilia B program, 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 hemophilia 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 hemophilia B gene therapy program 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 hemophilia could dramatically change not only the lives of patients but also the current hemophilia market that is dominated by protein replacement therapies.”