VYVDURA Approved in Japan for Chronic Inflammatory Demyelinating Polyneuropathy
核心洞察
Japan's MHLW has approved VYVDURA (efgartigimod alfa and hyaluronidase-qvfc (搜索)) for treating adults with Chronic Inflammatory Demyelinating Polyneuropathy (搜索) (CIDP (搜索)).
VYVDURA is the first and only neonatal Fc receptor (FcRn (搜索)) blocker approved for CIDP (搜索) treatment, administered as a weekly subcutaneous injection.
The approval is based on the ADHERE study, which demonstrated a 61% reduction in relapse risk compared to placebo (p<0.0001) and clinical improvement in 69% of patients.
Japan's Ministry of Health, Labour and Welfare (MHLW) has granted regulatory approval to VYVDURA (efgartigimod alfa and hyaluronidase-qvfc (搜索)) for the treatment of adults with chronic inflammatory demyelinating polyneuropathy (搜索) (CIDP (搜索)). This approval marks a significant advancement in the treatment landscape for CIDP, offering patients a novel therapeutic option with a convenient administration route. VYVDURA is co-formulated with Halozyme's ENHANZE® drug delivery technology.
VYVDURA is now approved for CIDP (搜索) as a once-weekly subcutaneous injection, administered within 30-90 seconds, and can be self-administered at home. This marks it as the first and only neonatal Fc receptor (FcRn (搜索)) blocker approved for the treatment of CIDP.
Clinical Evidence from the ADHERE Study
The MHLW approval is based on the ADHERE study, the largest clinical trial to date studying CIDP (搜索). The ADHERE study demonstrated that 69% (221/322) of patients treated with VYVDURA, regardless of prior treatment, showed evidence of clinical improvement, including improvements in mobility, function, and strength. The study met its primary endpoint (p<0.0001), demonstrating a 61% reduction (HR: 0.39 95% CI: 0.25; 0.61) in the risk of relapse versus placebo. Ninety-nine percent of trial participants elected to participate in the ADHERE+ open-label extension. The safety results were generally consistent with the known safety profile of VYVDURA in previous clinical studies and real-world use.
Luc Truyen, M.D., Ph.D., chief medical officer of argenx, stated, "With Vyvdura, CIDP (搜索) patients in Japan now have access to a novel therapy with a focused mode of action offering a convenient 30-to-90 second at-home self-injection option with an established efficacy and safety profile, as demonstrated by the ADHERE trial and real-world evidence."
Impact on CIDP Treatment
CIDP (搜索) is a progressive, immune-mediated rare and debilitating neuromuscular disorder of the peripheral nervous system. Patients experience a range of disabling mobility and sensory issues, including trouble standing from a seated position, pain and fatigue, and frequent tripping or falling. Many patients become wheelchair bound and are unable to work as the disease progresses. Currently, 85% of patients require ongoing treatment and nearly 88% of treated patients experience residual impairment and disability.
About VYVDURA
VYVDURA 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 (搜索)), VYVDURA results in the reduction of circulating IgG. It is the first-and-only approved FcRn blocker administered by subcutaneous injection for the treatment of CIDP (搜索).
VYVDURA was also approved by the MHLW for manufacturing and marketing in January 2024 and launched in April 2024 for the treatment of generalized myasthenia gravis (搜索) (gMG (搜索)). In March 2024, VYVDURA was designated as an Orphan Drug for the treatment of CIDP (搜索) by the MHLW.
Helen Torley, president and chief executive officer of Halozyme, commented, "We are pleased that VYVDURA, with our innovative ENHANZE drug delivery technology, is now approved for two indications in Japan, enabling greater flexibility and optionality for generalized myasthenia gravis (搜索) and CIDP (搜索) patients."