Efgartigimod/Hyaluronidase

近日，美国食品药品监督管理局（FDA）正在考虑对Argenx公司的皮下FcRn抑制剂Vyvgart Hytrulo采取监管行动。此前，Vyvgart Hytrulo被报告存在严重安全问题，特别是与慢性炎症性脱髓鞘多发性神经病（CIDP）的严重恶化相关。目前，FDA尚未公布具体的监管措施，但这一消息已经引起了市场的广泛关注。Argenx公司的股价在消息传出后有所下跌。投资者和患者都在密切关注FDA的最终决定，以及该决定对Vyvgart Hytrulo未来发展的潜在影响。该药物是一种FcRn拮抗剂，于2021年12月其静脉输注制剂首次获批。今年4月，VYVGART®Hytrulo(艾加莫德预充式皮下注射剂)美国获批治疗全身性重症肌无力 (gMG) 和慢性炎性脱髓鞘性多发性神经根神经病（CIPD）。Argenx公司作为一家专注于免疫学领域的生物制药企业，其Vyvgart Hytrulo药物曾被寄予厚望。该药物不仅在CIDP和gMG治疗领域有所突破，还在评估其治疗多种严重自体免疫性疾病的疗效。然而，此次FDA的监管行动可能会对该药物的市场前景产生重大影响。结束语：此次FDA的评估，源于对Vyvgart Hytrulo在CIDP治疗中安全性的担忧。尽管此前的临床研究ADHERE数据显示，该药物在治疗CIDP方面表现出一定的疗效，如69%的患者确认出现临床改善，但近期报告的严重安全问题促使FDA重新审视其安全性，并考虑是否需要采取监管措施。参考资料：FDA

iStock, Lemon_tm The safety update for Vyvgart rattled argenx’s shares on Monday, a reaction that analysts at William Blair said was “overdone.” The FDA is currently “evaluating the need for regulatory action” on argenx ’s Vyvgart Hytrulo after detecting a “severe” safety signal. The agency on Monday issued its quarterly update to the Adverse Event Reporting System, flagging the risk of severe worsening of chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) with Vyvgart Hytrulo. The drug, a neonatal Fc receptor blocker delivered via an injection under the skin, is approved to treat that disease as well as generalized myasthenia gravis (gMG). The safety update triggered share volatility for argenx, which was down by as much as 8% intraday on Monday, according to analysts from William Blair. The analysts called the stock reaction “overdone” in a note to clients issued the same day. Safety considerations for Vyvgart Hytrulo, they added, are not new in the prescribing community. “Yes, there is perceived risk of potential labeling language changes pending FDA review,” William Blair noted, “but we believe the more important aspect is clinician education on switching”—something that the company is already doing, according to the analysts. The Vyvgart franchise—which includes Vyvgart Hytrulo and Vyvgart, the original formulation of the same drug that is infused intravenously—is argenx’s only commercial presence, but one that analysts have high hopes for. In its note on Monday, William Blair said that despite the safety flags from the FDA, Vyvgart still has “multiblockbuster potential” in CIDP and gMG. In 2024, Vyvgart products brought in $2.2 billion. Argenx is continuing to develop its Vyvgart line with studies in other indications, such as thyroid eye disease, Sjogren’s disease, lupus nephropathy and myositis. This last indication, in particular, could open “significant opportunity” for the franchise, according to William Blair. Still, Vyvgart’s earnings could be put at risk in coming years as the drug approaches its loss of exclusivity, which could come as early as 2033 . In preparation, the biotech is advancing a pipeline of investigational therapies, anchored by the antibody empasiprubart for dermatomyositis, multifocal motor neuropathy (MMN), delayed graft function and CIDP. William Blair sees “billion-dollar opportunities” for argenx’s pipeline, particularly for empasiprubart in MMN, the firm’s analysts wrote on Monday. On Tuesday, argenx further deepened its pipeline, betting $1.5 billion on a partnership with Unnatural Products to develop oral macrocyclic peptides for “undruggable”—but not yet specified—disease targets.