美国首款质粒DNA免疫疗法获加速批准：FDA放行 Voceryq 治疗罕见气道疾病，为患者减少反复手术
美国食品药品监督管理局（FDA）10月30日授予 Voceryq 加速批准，用于治疗成人复发性呼吸道乳头状瘤病（RRP）。这是美国首个获批的质粒 DNA 免疫疗法，为治疗选择有限的患者群体提供了一种从病源入手、而不只是处理症状的新疗法。
复发性呼吸道乳头状瘤病是一种由人乳头瘤病毒（HPV）引起的罕见疾病，会在患者气道内形成小的疣状增生。成人患者往往要经历反复手术切除增生的疲惫循环，始终难以摆脱疾病。Voceryq 的完整药品名为 doruxapapogene ralaplasmid 与 lalikinogene sifuplasmid-gxfd，它调动人体自身的免疫系统对抗 RRP 的根本病因，已显示可减轻手术干预的负担。
FDA 生物制品评价与研究中心主任 Karim Mikhail 表示：「今天的批准代表着一项有意义的科学突破：一种质粒 DNA 免疫疗法，利用人体自身的免疫系统对抗 RRP 的根本原因。对患者来说，这可能意味着更少的手术、更少的生活打扰，以及更多按自己意愿生活的时光。」
FDA Grants Accelerated Approval to Voceryq, the First Plasmid DNA-Based Immunotherapy in the U.S., for a Rare Airway Disease
The U.S. Food and Drug Administration on October 30 granted accelerated approval for Voceryq (doruxapapogene ralaplasmid and lalikinogene sifuplasmid-gxfd) for the treatment of adults with recurrent respiratory papillomatosis (RRP), a rare disease caused by human papillomavirus (HPV) that leads to small, wart-like growths in the airways. It is the first approval of a plasmid DNA-based immunotherapy in the United States, offering patients a new option that targets the root cause of RRP rather than only its symptoms and has been shown to reduce the burden of surgical intervention.
Adults living with RRP often face an exhausting cycle of repeated surgeries, managing the disease without ever truly escaping it. The new therapy harnesses the body's own immune system against the underlying cause.
"Today's approval represents a meaningful scientific breakthrough: a plasmid DNA-based immunotherapy that harnesses the body's own immune system to fight the underlying cause of RRP," said Karim Mikhail, Director of the Center for Biologics Evaluation and Research. "For patients, that could mean fewer surgeries, fewer disruptions, and more time living their lives on their own terms."
来源：GSM·美国（原文链接）
图片来源：图： FDA 国家毒理研究中心实验室。美国食品药品监督管理局，公共领域。 / A laboratory at the FDA's National Center for Toxicological Research. U.S. Food and Drug Administration, public domain.
