AMT Receives Orphan Designation in the European Union for Hemophilia B Gene Therapy - Gilde Healthcare

AMT Receives Orphan Designation in the European Union for Hemophilia B Gene Therapy

November 16, 2011

Amsterdam, The Netherlands – 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.”

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.

Gilde Healthcare company Moximed reports treatment of first commercial patients post FDA approval

Moximed, a medical device company on a mission to improve the standard of care for people with knee osteoarthritis (OA), today announced the first commercial cases of the MISHA™ Knee System, an implantable shock absorber...
September 6, 2023

Gilde Healthcare company Withings launches its next-generation scanwatch with enhanced sensors to monitor additional health metrics 24/7

In 2014, Withings, a pioneer of the digital health movement, announced the world’s first hybrid watch. Today it furthers its leadership of the hybrid connected watch sector, unveiling the latest generation of its groundbreaking ScanWatch....
August 31, 2023

Gilde Healthcare company Big Health expands into adolescent mental health through acquisition of Limbix

Big Health, the leading provider of non-drug digital treatments for mental health, has acquired Limbix, creators of the first, evidence-based digital therapeutic for teens and young adults with symptoms of depression. Since its founding in...
July 17, 2023