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AMT's Hemophilia B Gene Therapy Program Granted Orphan Drug Status

February 1, 2012

Amsterdam Molecular Therapeutics (AMT), a company specializing in human gene-based therapy, announced recently that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its gene therapy program for the treatment of hemophilia B. The FDA’s Office of Orphan Products Development grants orphan drug status, which means the drugs go through an expedited review process. The designation allows for accelerated approval, so that much-needed drugs or biologics become available to patients with rare, life-threatening diseases who need effective treatments. Orphan designation for AMT’s hemophilia program in the European Union was granted in November 2011.

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 in Memphis, TN, and the University College London (UCL). In December 2011, the results of a breakthrough hemophilia B gene therapy study on six patients conducted by St. Jude’s and UCL were published in the New England Journal of Medicine.

According to a January 4th press release AMT is preparing for additional clinical development work to establish safety, tolerability and proof-of-concept with a factor IX gene therapy product using its proprietary AAV production system.

“U.S. orphan designation provides additional support for our hemophilia B gene therapy program and supplements the designation in the EU received in November,” said Jörn Aldag, CEO of AMT. “The early clinical success seen with the program to date by our partners is very encouraging. We will build on this success in the coming months.”

Source: AMT press release dated January 4, 2012