공시 • May 11
argenx SE Announces U.S. FDA Approval Expanding VYVGART and VYVGART Hytrulo for Use in All Adult Patients Living with gMG
argenx SE announced the U.S. Food and Drug Administration approved a label expansion for VYVGART (efgartigimod alfa-fcab) and VYVGART Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) for the treatment of adult patients with generalized myasthenia gravis (gMG). The approved supplemental Biologics License Application expands VYVGART’s indication to include all serotypes of adult patients living with gMG – anti-AChR-Ab positive, anti-MuSK-Ab positive, anti-LRP4-Ab positive, and triple seronegative. The approval is based on data from the Phase 3 ADAPT SERON study, the study to date of patients with gMG who do not have detectable anti-acetylcholine receptor antibodies (AChR-Ab) across three serotypes – anti-MuSK-Ab positive, anti-LRP4-Ab positive, and triple seronegative. The overall population of patients in the study treated with VYVGART showed rapid, significant and sustained improvements in their gMG symptoms, including speech, vision, physical function and swallowing, among others. In addition, VYVGART was well tolerated across serotypes, with safety consistent with the established profile in patients with anti-AChR-Ab positive gMG. Detailed results from the Phase 3 ADAPT SERON study: Patients showed clinically meaningful improvements in disease activity across all three serotypes – anti-MuSK-Ab positive, anti-LRP4-Ab positive, and triple seronegative. The primary endpoint was met (p=0.0068), demonstrating that patients treated with VYVGART achieved a statistically significant improvement in MG-ADL (Myasthenia Gravis Activities of Daily Living) total score compared to placebo at week 4. In the overall population – across all serotypes in the study – mean change from baseline in patients treated with VYVGART was a clinically meaningful 3.35-point improvement in MG-ADL total score at week 4. Improvements in MG-ADL and Quantitative Myasthenia Gravis (QMG) scores were observed across subsequent treatment cycles in the overall population and in all serotypes studied. VYVGART was well tolerated across serotypes, with safety consistent with the established profile in patients with anti-AChR-Ab positive gMG. Argenx recently announced positive top-line results from the ADAPT OCULUS study of VYVGART Hytrulo in ocular MG and remains focused on advancing the development of VYVGART for all patients living with MG, including pediatric gMG patient populations in the ADAPT Jr study. VYVGART is available to patients in three administration options, including VYVGART Hytrulo self-injection with a prefilled syringe. The argenx patient support program, My VYVGART Path, can help patients and healthcare providers navigate access. My VYVGART Path resources include disease and product education, access support and benefits verification, and financial assistance programs for eligible patients. Generalized myasthenia gravis (gMG) is a rare, chronic, neuromuscular autoimmune disease caused by pathogenic IgGs targeting the neuromuscular junction (NMJ), resulting in impaired neuromuscular transmission and debilitating and potentially life-threatening muscle weakness and chronic fatigue. Approximately 80% of patients with gMG have detectable antibodies against the AChR in sera, and these patients are diagnosed as AChR-Ab positive gMG. Approximately 20% of patients with gMG do not have detectable serum antibodies directed against AChR and are referred to as having anti-AChR antibody negative gMG. These patients may have detectable autoantibodies targeting other NMJ proteins, such as muscle-specific tyrosine kinase (MuSK) and low-density lipoprotein receptor-related protein 4 (LRP4), or others. Anti-MuSK antibodies are detected in approximately 1-10% of patients with gMG, while anti-LRP4 antibodies are detected in approximately 1-5% of patients with gMG. About 10% of patients do not have any detectable autoantibodies against AChR, MuSK or LRP4. These triple seronegative patients have historically been excluded from studies and have a higher disease burden and unmet medical need compared to patients with detectable autoantibodies. The Phase 3 ADAPT SERON study is a randomized, double-blind, placebo-controlled, multi-center study evaluating the safety and efficacy of efgartigimod in adults with anti-AChR antibody negative gMG (n=119) across North America, Europe, China, and the Middle East. Part A randomized participants (1:1) received 4 once-weekly infusions of efgartigimod IV or placebo, followed by a 5-week follow-up. Part B is an open-label period: participants receive 2 fixed cycles of 4 once-weekly efgartigimod infusions (4-week interval between cycles); from cycle 3 onward, additional cycles could be started =1 week after the last administration of the previous cycle, based on clinical status. The primary endpoint is the MG-ADL total score change from baseline to week 4 (day 29) in part A. Other scales of evaluation include QMG, MG-QoL 15r, MGC, and EQ-5D-5L VAS. Enrolled participants had a confirmed MG diagnosis by an independent panel of experts, and an MG-ADL total score of 5 or greater. Participants were on a stable dose of at least one gMG treatment prior to randomization, including acetylcholinesterase inhibitors, corticosteroids or nonsteroidal immunosuppressive drugs. Participants were eligible to enroll in ADAPT SERON if they were anti-AChR antibody negative gMG, which included participants who are anti-MuSK-Ab positive, anti-LRP4-Ab positive, and triple seronegative. MG-ADL is a validated measure of disease activity in patients living with MG, which evaluates the functional impact of symptoms on daily activities such as speaking, chewing, swallowing, breathing, and limb strength. VYVGART and VYVGART HYTRULO are both prescription medicines used to treat adults with generalized myasthenia gravis (gMG). It is not known if VYVGART or VYVGART HYTRULO is safe and effective in children. Do not take VYVGART if allergic to efgartigimod alfa or any of the ingredients in VYVGART.
