Acceptance by China’s NMPA of the BLA for efgartigimod alfa injection (subcutaneous injection) for patients with generalized myasthenia gravis

The BLA submission is supported by positive results from the global Phase III ADAPT-SC study, demonstrating noninferior total IgG reduction from baseline at day 29 with SC administered efgartigimod compared to intravenous (IV) administered efgartigimod in adult patients with gMG. Patients treated with efgartigimod SC achieved mean total IgG reduction of 66.4% from baseline at day 29, compared to 62.2% reduction with efgartigimod IV.

The safety profile for efgartigimod SC was also consistent with the ADAPT study. It was generally well-tolerated; the most frequent adverse event being injection site reactions (ISRs), commonly observed with biologics administered subcutaneously. All ISRs were mild to moderate and resolved over time.

In June 2023, efgartigimod alfa injection (Vyvgart) for IV use was approved by China’s NMPA as an add on to standard therapy for the treatment of adult patients with gMG who are anti-acetylcholine receptor (AChR) antibody positive. In June 2023, Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) was approved by the FDA for SC use for the treatment of adult patients with gMG who are anti-AChR antibody positive.

Vyvgart is the first approved FcRn blocker in the United States, EU and China for the treatment of adults with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive, and in Japan for the treatment of adults with gMG who do not have sufficient response to steroids or non-steroidal immunosuppressive therapies (ISTs).