4 January 2012 - Dutch human gene therapy specialist Amsterdam Molecular Therapeutics (AMS:AMT) said today its gene therapy programme for the treatment of hemophilia B had obtained US Food and Drug Administration (FDA) Orphan Drug status.

The company's hemophilia programme was granted orphan designation in the EU in November last year.

The hemophilia B programme, which consists of an adeno-associated viral (AAV) vector containing the human factor IX gene, is being evaluated in a Phase I/II trial.

Encouraging data from an initial 6 patients, recently published in the New England Journal of Medicine, demonstrates that gene therapy administration resulted in a reduced need for protein replacement treatment, the standard care for hemophilia patients.

The firm said it 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.