ARGX - argenx Announces FDA Acceptance of Supplemental Biologics License Application with Priority Review for VYVGART Hytrulo in Chronic Inflammatory Demyelinating Polyneuropathy | Benzinga
Prescription Drug User Fee Act (PDUFA) target action date is June 21, 2024
If approved, VYVGART® Hytrulo will be the first neonatal Fc receptor (FcRn) blocker to treat CIDP
February 20, 2024, 7:00 AM CET
Amsterdam, the 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 ...