Argenx advised by Japan regulator on Phase 3 myasthenia gravis trial

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 it has received feedback from the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan on the design of a Phase 3 trial and regulatory pathway towards potential marketing authorization of efgartigimod (ARGX-113) in patients with generalized myasthenia gravis (gMG).

Based on feedback from the PMDA, argenx expects the data from the planned global Phase 3 registration trial, if positive, will serve as the basis to submit for marketing authorization in Japan.

“Japan represents a very important potential market for us and one we see as a top priority as we advance the development of efgartigimod in gMG patients globally. There remains a significant unmet need in Japan for new treatment options for MG, and we are grateful for the feedback from the PMDA in facilitating a regulatory path towards achieving this goal,” commented Keith Woods, Chief Operating Officer of argenx. “We look forward to launching the global Phase 3 trial of efgartigimod in gMG before the end of the year, including in sites across Japan, and continuing the ongoing discussions with the PMDA as we map out a potential path to market for our drug candidate, if approved.”

The global Phase 3 trial expects to enroll approximately 150 patients with gMG, including patients from Japan as well as North America and Europe. The trial will be placebo-controlled and will evaluate the efficacy over 26 weeks of a 10 mg/kg dose of efgartigimod in approximately 150 gMG patients, including both acetylcholine receptor (AChR) autoantibody positive and AChR autoantibody negative patients whose disease is driven primarily by MuSK and LRP4 autoantibodies. In addition, patients can roll over into an open-label extension study for a period of one year.

argenx plans to initiate the global Phase 3 registration trial of efgartigimod in gMG before the end of 2018. Data from the trial, if positive, may also serve as the basis for a Biologics License Application (BLA) in the U.S. based on feedback from the U.S. Food and Drug Administration (FDA).

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