Publication date: 25/09/2017
argenx (Euronext & Nasdaq: ARGX), a clinical-stage biotechnology company developing a deep pipeline of differentiated antibody-based therapies for the treatment of severe autoimmune diseases and cancer, today announced that the Office of Orphan Products Development of the U.S. Food and Drug Administration (FDA) has granted orphan status for the use of ARGX-113 for the treatment of myasthenia gravis.
MG is an autoimmune disorder associated with muscle weakness that is triggered by IgG auto-antibodies. These antibodies attack critical signalling proteins at the junction between nerve and muscle cells, thereby impairing their communications signals. There are limited effective and sustainable treatments for MG. ARGX-113 has the potential to eliminate patient symptoms while minimizing common side effects seen with current treatments by reducing the pathogenic IgG levels.
About orphan drug designation
Orphan drug status is granted by FDA to a drug or biological product to treat a rare disease or condition upon request of a sponsor. Orphan drug designation qualifies for various development incentives, including tax credits for qualified clinical testing, a waiver from FDA's application User Free for marketing application, and a 7-year period of marketing exclusivity in the US.
The granting of an orphan designation request does not alter the standard regulatory requirements and process for obtaining marketing approval. Safety and effectiveness of a drug must be established through adequate and well-controlled studies.
ARGX-113 is an investigational therapy for treatment of IgG-mediated autoimmune diseases. ARGX-113 is the Fc-portion of an antibody that has been modified by the argenx proprietary ABDEG(TM) technology to increase its affinity for FcRn beyond that of normal IgG antibodies. As a result, ARGX-113 blocks antibody recycling and leads to fast depletion of the autoimmune disease-causing IgG autoantibodies. The development work on ARGX-113 is done in close collaboration with Prof. E. Sally Ward (University of Texas Southwestern Medical and Texas A&M University Health Science Center, a part of Texas A&M University (TAMHSC)).
argenx is a clinical-stage biotechnology company developing a deep pipeline of differentiated antibody-based therapies for the treatment of severe autoimmune diseases and cancer. We are focused on developing product candidates with the potential to be either first-in-class against novel targets or best-in-class against known, but complex, targets in order to treat diseases with a significant unmet medical need. Our ability to execute on this focus is enabled by our suite of differentiated technologies. Our SIMPLE Antibody(TM) Platform, based on the powerful llama immune system, allows us to exploit novel and complex targets, and our three antibody engineering technologies are designed to enable us to expand the therapeutic index of our product candidates. www.argenx.com