Amsterdam, Netherlands – argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, today announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) approved VYVGART (efgartigimod alfa) for intravenous (IV) use in adults with primary immune thrombocytopenia (ITP).
“argenx is on a mission to deliver transformative medicines for people living with severe autoimmune disease,” said Tim Van Hauwermeiren, Chief Executive Officer of argenx. “We have always believed VYVGART has the potential to treat many IgG-mediated autoimmune diseases, and now patients in Japan, who have been waiting for a new treatment option, have one for ITP. My sincere thanks to all of those who contributed to today’s milestone approval in Japan – it takes true collaboration to bring real innovation for the millions of patients around the world living with rare autoimmune disease. And our work has only just begun.”
“ITP is a severe, debilitating autoimmune disease, and ITP patients typically experience a significantly lower quality of life,” said Luc Truyen, M.D., Ph.D., Chief Medical Officer of argenx. “Physicians now treat ITP via a trial-and-error approach – or by cycling through current therapies. By reducing circulating autoantibodies, VYVGART is uniquely designed to serve as a precision intervention that targets the underlying disease biology of ITP. We are grateful to all of those who contributed to advancing our understanding of the disease so that we could bring VYVGART to patients in Japan.”
The approval of VYVGART is based on results from the global Phase 3 ADVANCE-IV trial, which were published in the September 2023 issue of The Lancet. ADVANCE successfully met its primary endpoint, demonstrating that a higher proportion of chronic ITP patients receiving VYVGART achieved a sustained platelet count response compared to placebo. VYVGART demonstrated rapid onset of effect in chronic and persistent ITP patients, as well as a 51% response rate on the International Working Group (IWG) score,
which is a measure developed by the world’s leading experts on ITP and highly relevant to clinical care. Primary endpoint responders were observed across patient types regardless of age, disease severity, time since diagnosis, prior ITP treatment or background medication. VYVGART was well-tolerated in this 24-week study and the observed safety and tolerability profile was consistent with previous clinical trials.
About Immune Thrombocytopenia
Immune thrombocytopenia (ITP) is an autoimmune disorder where immunoglobulin G (IgG) autoantibodies destroy platelets and reduce platelet production, which can lead to an increased risk of excessive bleeding and bruising. In severe cases, frequent bleeding events can cause anemia or even brain hemorrhage in rare cases. ITP is also associated with debilitating fatigue and significant impacts on mental health, including anxiety, fear and depression. Many ITP patients are inadequately controlled on current therapies so there remains a significant unmet need for additional treatment options.
About Phase 3 ADVANCE Trial
The ADVANCE trial enrolled 131 adult patients with chronic and persistent ITP. Patients were heavily pretreated and 67% of patients had received three or more prior ITP therapies, including 59% who had prior thrombopoietin receptor agonist (TPO-RAs) experience, 34% with prior rituximab experience and 37% with a history of splenectomy. Patients were insufficiently controlled at baseline with mean platelet counts of 17×109/L across all patients. Of patients who completed the full ADVANCE study, 94% (63/67) of VYVGART-treated patients and 97% (38/39) of placebo patients continued to the ADVANCE+ open-label extension study.
See the full Prescribing Information for VYVGART in the U.S., which includes the below Important Safety Information. For more information related to VYVGART in Japan, visit: argenx.jp.
About VYVGART® (efgartigimod alfa-fcab)
VYVGART is a prescription medicine used to treat a condition called generalized myasthenia gravis, which causes muscles to tire and weaken easily throughout the body, in adults who are positive for antibodies directed toward a protein called acetylcholine receptor (anti-AChR antibody positive).
About argenx
argenx is a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases. Partnering with leading academic researchers through its Immunology Innovation Program (IIP), argenx aims to translate immunology breakthroughs into a world-class portfolio of novel antibody-based medicines. argenx developed and is commercializing the first approved neonatal Fc receptor (FcRn) blocker in the U.S., Japan, Israel, the EU, the UK, Canada and China. The Company is evaluating efgartigimod in multiple serious autoimmune diseases and advancing several earlier stage experimental medicines within its therapeutic franchises.
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