04-01-12
Amsterdam Molecular Therapeutics Receives US Orphan Designation for Hemophilia B Gene Therapy

Amsterdam, The Netherlands – January 4, 2011 – Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today 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. Orphan designation in the U.S. could provide up to seven years market exclusivity on regulatory approval. 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 (Memphis, USA) and University College London (UK). Promising data from an initial 6 patients, recently published in the New England Journal of Medicine (N Engl J Med 2011; 365:2357-2365), 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.

“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.”

About Amsterdam Molecular Therapeutics

AMT is a world leader in the development of human gene based therapies. AMT has a product pipeline of gene therapy products in development for hemophilia B, acute intermittent porphyria, Parkinson’s disease and SanfilippoB. Using adeno-associated viral (AAV) derived vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate probably the world’s first stable and scalable AAV manufacturing platform. This proprietary platform can be applied to a large number of rare (orphan) diseases caused by one faulty gene and allows AMT to pursue its strategy of focusing on this sector of the industry.  AMT was founded in 1998 and is based in Amsterdam. Further information can be found at www.amtbiopharma.com.

Source: Amsterdam Molecular Therapeutics
Biotech Company Profile
Amsterdam Molecular Therapeutics B.V. (AMT)
Meibergdreef 61, 1100 DA, Amsterdam
Phone: +31 (20) 5667394, www.amtbiopharma.com
Sector: Biotechnology - Therapeutics
Employees local: 90
Description: AMT aspires to use its know-how and expertise in gene therapy to develop innovative treatments that significantly improve the lives of patients with serious, debilitating diseases.AMT focuses on developing therapies for inherited diseases that affect small numbers of patients (orphan diseases). In addition, several of our programs allow us to target selected non-orphan diseases associated with serious morbidity and / or high mortality that can be treated with gene therapy.