Amsterdam, Netherlands – 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 U.S. Food and Drug Administration (FDA) has accepted for priority review a supplemental Biologics License Application (sBLA) for VYVGART Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) for the treatment of chronic inflammatory demyelinating polyneuropathy (CIDP). The application has been granted a PDUFA target action date of June 21, 2024.
“Today’s announcement brings us one step closer to delivering the transformative innovation of VYVGART Hytrulo to CIDP patients,” said Luc Truyen, Chief Medical Officer of argenx. “CIDP is yet another example of an autoimmune disease that has not been well understood, and for which there has been insufficient innovation for patients. We chose to use a priority review voucher to accelerate review of our submission because CIDP patients have long been waiting for new treatment options. FDA’s acceptance of the sBLA represents an important milestone in our continued drive to bring novel treatments for rare, autoimmune diseases, and a significant step forward for people whose lives have been profoundly impacted by this devastating disease.”
The sBLA is supported by data from the ADHERE study, the largest clinical trial of CIDP to date, evaluating the safety and efficacy of subcutaneously administered VYVGART Hytrulo in adults with CIDP. The study met its primary endpoint (p=0.000039), demonstrating a 61% lower risk of relapse (HR: 0.39 95% CI: 0.25; 0.61) with VYVGART Hytrulo compared to placebo. In the open-label Stage A of the study, 67% of patients showed evidence of clinical improvement (ECI) following treatment with VYVGART Hytrulo. Given the mechanism of action of VYVGART Hytrulo as an FcRn blocker, the clinical results established that IgG autoantibodies play a significant role in the underlying biology of CIDP.
VYVGART Hytrulo was well-tolerated with a safety profile that is consistent with prior clinical trials and the known profile of VYVGART®. After completing ADHERE, 99% of eligible patients (226/228) continued to the ADHERE-+ open-label extension study.
About Chronic Inflammatory Demyelinating Polyneuropathy
Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare and serious autoimmune disease of the peripheral nervous system. Although confirmation of disease pathophysiology is still emerging, there is increasing evidence that IgG antibodies play a key role in the damage to the peripheral nerves. People with CIDP experience fatigue, muscle weakness and a loss of feeling in their arms and legs that can get worse over time or may come and go. These symptoms can significantly impair a person’s ability to function in their daily lives. Without treatment, one-third of people living with CIDP will need a wheelchair.
About VYVGART® Hytrulo(efgartigimod alfa and hyaluronidase-qvfc)
VYVGART Hytrulo is a subcutaneous combination of efgartigimod alfa, a human IgG1 antibody fragment marketed for intravenous use as VYVGART®, and recombinant human hyaluronidase PH20 (rHuPH20), Halozyme’s ENHANZE® drug delivery technology to facilitate subcutaneous injection delivery of biologics. In binding to the neonatal Fc receptor (FcRn), VYVGART Hytrulo results in the reduction of circulating IgG. It is the first-and-only approved FcRn blocker administered by subcutaneous injection.
VYVGART Hytrulo is the proprietary name in the U.S. for subcutaneous efgartigimod alfa and recombinant human hyaluronidase PH20. It may be marketed under different proprietary names following approval in other regions.
About argenx
argenx is a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases. Partnering with leading academic researchers through its Immunology Innovation Program (IIP), argenx aims to translate immunology breakthroughs into a world-class portfolio of novel antibody-based medicines. argenx developed and is commercializing the first approved neonatal Fc receptor (FcRn) blocker in the U.S., Japan, Israel, the EU, the UK, Canada and China. The Company is evaluating efgartigimod in multiple serious autoimmune diseases and advancing several earlier stage experimental medicines within its therapeutic franchises.
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