argenx announces positive CHMP opinion for subcutaneous efgartigimod for generalized myasthenia gravis
argenx SE (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 EC approval of the SC injectable formulation 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. SC efgartigimod is formulated with Halozyme’s ENHANZE® drug delivery technology to facilitate subcutaneous delivery of biologics.
“Generalized myasthenia gravis is a complex and devastating disease that is debilitating for people who live with it, making routine movements exhausting and challenging to perform,” said Prof. dr. Jan De Bleecker, Ghent University Hospital and Ghent University. “I’m pleased to learn of the CHMP’s positive opinion as it represents a significant advancement for the gMG community who would benefit from an additional, effective treatment option that can improve quality of life and better manage this chronic condition. In particular, SC efgartigimod has the potential to have a positive impact on treatment convenience, leading to a broader positive impact for patients and healthcare systems.”
“The positive recommendation by the CHMP for the SC injectable formulation of efgartigimod brings us one step closer to broadening our treatment offering for people living with gMG in Europe,” said Anant Murthy, Ph.D., General Manager, EMEA, argenx. “Our mission is to transform the treatment of severe autoimmune disease, and we remain committed to providing gMG patients a second innovation that could further address treatment burden. We are particularly pleased with the possibility for self-administration of the SC formulation, which may provide additional treatment flexibility for physicians and patients.”
The CHMP recommendation is based on positive results from the Phase 3 ADAPT-SC study. The ADAPT-SC trial met its primary endpoint of noninferiority, where SC efgartigimod demonstrated a mean total IgG reduction of 66.4% from baseline at day 29, compared to 62.2% with the IV formulation. Additional key secondary endpoints were met, which were consistent with efficacy measures from the ADAPT IV study identifying the correlation between IgG reduction and clinical benefit in gMG.
SC efgartigimod has a demonstrated safety profile, consistent with the ADAPT IV clinical trial with the exception of injection site reactions (ISRs), which are commonly observed with biologics administered subcutaneously. ISRs were mild to moderate and did not lead to treatment discontinuation.
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 SC efgartigimod. The EC is expected to make a decision within approximately 60 days following CHMP recommendation. The decision will apply to all 27 European Union Member States, and also to Iceland, Norway and Liechtenstein.