Approval of efgartigimod alfa injection (subcutaneous injection) for generalized myasthenia gravis in China – Zai Lab + argenx

Zai Lab Limited and argenx announced that China’s National Medical Products Administration (NMPA) approved the Biologics License Application (BLA) for efgartigimod alfa injection (subcutaneous injection) (efgartigimod SC), 1,000mg (5.6ml)/vial indicated 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.

“We are pleased to receive NMPA approval for efgartigimod SC, marking an important milestone as we bring another first-in-class option to gMG patients in China,” said Rafael G. Amado, M.D., President, Head of Global Research and Development at Zai Lab. “The addition of a new treatment option for gMG patients enhances flexibility for patients, potentially further simplifying the regimen and making therapy more accessible within the community. We appreciate the NMPA for their thorough assessment and recognition of the therapy’s differentiated profile and the large unmet medical need in China.”

“The NMPA approval for efgartigimod SC is yet another key milestone on our journey to expand into new patient populations around the world with our transformative medicine,” said Tim Van Hauwermeiren, Chief Executive Officer of argenx. “We celebrate this achievement with our partner, Zai Lab, who shares our mutual passion for bringing needed innovation to patients with gMG in China. We are impressed by the team’s incredible launch execution, bringing 2,700 new patients onto Vyvgart IV treatment in the first quarter of 2024, which only underscores the high unmet need that remains for gMG patients. The addition of a flexible 30-to-90 second subcutaneous injection opens the door for new patients in China, while taking into account personal preference and convenience. We look forward to continuing our partnership and expanding our footprint in one of the world’s fastest growing markets to reach more people living with severe autoimmune diseases.”

“There are approximately 170,000 people living with gMG in China,” said Prof. Xueqiang Hu, M.D., Ph.D., Chief Physician of Department of Neurology, the Third Affiliated Hospital of Sun Yat-sen University. “Compared to fixed infusion schedules, the availability of efgartigimod SC allows a more individualized and flexible treatment approach based on patient needs without sacrificing clinical benefit or safety. In the global Phase III ADAPT-SC study, efgartigimod SC demonstrated consistent benefit and safety compared to the intravenous product. This is a meaningful advancement for the patient community, and we are grateful to Zai Lab for supporting patients who have been devastated by this disease for so long.”

The BLA approval is supported by positive results from the global Phase III ADAPT-SC study, a bridging study to the Phase III ADAPT study, which formed the basis for approval of intravenous Vyvgart in adult gMG patients. In the ADAPT-SC study, the primary endpoint of noninferiority was met (p< 0.0001), and efgartigimod SC demonstrated mean total IgG reduction of 66.4% from baseline at day 29, compared to 62.2% with efgartigimod IV. Additional key secondary endpoints were also met, which were consistent with efficacy measures from the ADAPT study identifying the correlation between total IgG reduction and clinical benefit in gMG.