argenx Receives Positive CHMP Opinion for Efgartigimod for the Treatment of Adult Patients with Generalized Myasthenia Gravis in Europe

• Positive opinion based on Phase 3 ADAPT trial showing efgartigimod provided clinically meaningful improvements in strength and quality of life measures
  
  
• European Commission decision on marketing authorization application (MAA) expected in approximately 60 days
  
  
• If approved, efgartigimod will be the first neonatal Fc receptor (FcRn) blocker for the treatment of adults in Europe living with rare neuromuscular disease generalized myasthenia gravis (gMG)
  
  

Breda, the Netherlands—June 24, 2022—argenx (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended European Commission (EC) approval of efgartigimod as an add-on to standard therapy for the treatment of adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive.

The positive CHMP opinion is a scientific recommendation for marketing authorization, serving as a basis for the EC’s final decision on argenx’s application for efgartigimod. The EC is expected to make a decision within approximately 60 days following CHMP recommendation, which will be applicable to all 27 European Union Member States, in addition to Iceland, Norway and Liechtenstein.

“The current treatment of generalized MG in Europe leaves many patients with insufficiently controlled symptoms that markedly decrease quality of life. Caregivers and medical teams are well aware of the need for new treatment options that are safe, effective and targeted to the disease biology” said Anthony Béhin, MD neuromuscular physician at Institut de Myologie, La Pitié Salpétrière, Paris. “As a practicing neurologist regularly seeing people who live with this debilitating chronic disease, the CHMP’s positive opinion of efgartigimod represents an exciting advancement toward bringing a new treatment option to these patients in Europe.”

The MAA included results from the pivotal Phase 3 ADAPT trial, which were published in the July 2021 issue of The Lancet Neurology. The ADAPT trial met its primary endpoint, demonstrating that significantly more anti-acetylcholine receptor (AChR) antibody positive gMG patients were responders on the Myasthenia Gravis Activities of Daily Living (MG-ADL) scale following treatment with efgartigimod compared with placebo (68% vs. 30%; p<0.0001). Responders were defined as having at least a two-point reduction on the MG-ADL scale sustained for four or more consecutive weeks during the first treatment cycle.