FDA批准首个基于质粒DNA的免疫疗法用于成人复发性呼吸道乳头状瘤病
正文 · 中文译文
FDA新闻稿 FDA批准首个基于质粒DNA的免疫疗法用于成人复发性呼吸道乳头状瘤病 突破性疗法提供额外治疗选择，从疾病根源而非仅症状进行对抗 更多新闻公告 即时发布： 2026年10月30日 美国食品药品监督管理局（FDA）今日授予Voceryq（doruxapapogene ralaplasmid与lalikinogene sifuplasmid-gxfd）加速批准，用于治疗成人复发性呼吸道乳头状瘤病（RRP），这是一种由人乳头瘤病毒（HPV）引起的罕见疾病，可导致患者气道内形成小的疣样生长物。该批准是美国首个基于质粒DNA的免疫疗法获批，为患者提供了一种新的治疗选择，其靶向RRP的根本病因，并已显示可减轻手术干预负担，标志着为治疗选择有限的患者群体迈出了重要一步。 “患有复发性呼吸道乳头状瘤病的成年人常常面临一个令人疲惫且无休止的循环：反复接受手术、管理疾病，却始终无法真正摆脱它。今天的批准代表了一项有意义的科学突破：一种基于质粒DNA的免疫疗法，利用人体自身的免疫系统来对抗复发性呼吸道乳头状瘤病（RRP）的根本病因，”生物制品评价与研究中心（CBER）主任、药学学士、理学硕士Karim Mikhail表示。“对患者而言，这可能意味着更少的手术、更少的生活中断，以及更多按自己意愿生活的时间。FDA很自豪能帮助让这一可能性变得触手可及。” 该机构将此加速批准建立在一项临床试验数据的基础上，该数据显示Voceryq有助于降低成年RRP患者为切除生长物所需外科手术的频率——这是一个合理可能预测临床获益的中间临床终点。加速批准 pathway 允许FDA基于对合理可能预测临床获益的替代终点或中间临床终点的效应，批准用于严重或危及生命疾病的药物或生物制品。生产商必须开展批准后临床试验，以验证并描述Voceryq的临床获益。 复发性呼吸道乳头状瘤病（RRP）是一种罕见的慢性疾病，由HPV 6型和/或11型引起的疣状生长物在气道内形成，最常见于喉部（声盒）周围，尤其是声带。这些生长物称为乳头状瘤，可引起声音嘶哑、慢性咳嗽和声音改变等症状，严重时可蔓延至呼吸道其他部位，导致呼吸困难。虽然手术可以切除这些生长物，但该病无法治愈，即使生长物被切除后病毒仍持续存在于组织中，因此患者往往需要多年反复接受手术。在某些病例中，RRP随时间推移存在癌变风险。 Voceryq是一种基于重组质粒DNA的免疫疗法，将两种质粒（最初来源于细菌的小片段DNA）递送至机体细胞内，其中一种质粒含有特定HPV蛋白的遗传指令，另一种质粒含有称为IL-12的免疫应答增强分子。该疗法旨在刺激免疫系统对由引起RRP的特定类型HPV（6型和11型）感染的细胞作出应答并攻击这些细胞。Voceryq通过肌内（IM）注射使用CELLECTRA 5PSP装置给药，该装置递送剂量并促进电穿孔，电穿孔是一种利用短暂电脉冲暂时且可逆地在细胞膜上形成孔隙的技术，从而使Voceryq能够递送至机体细胞内。 Voceryq 的安全性和有效性在一项开放标签多中心临床试验中进行了评估，该试验纳入 32 名诊断为复发性呼吸道乳头状瘤病（RRP）的成人患者。符合条件的患者在 Voceryq 治疗前 12 个月内接受过两次或以上喉气管乳头状瘤切除术，包括试验入组时计划进行的手术。在至少接受过三次基线手术的 26 名患者中，有 7 名（27%）在治疗后第一年内不需要任何手术干预。 Voceryq 最常见报告的不良反应为注射部位反应、头痛、疲劳、高血压（血压升高）、恶心和头晕。与电穿孔技术相关的不良反应可能包括短暂肌肉收缩或手足抽搐、给药部位暂时性出血、焦虑、血管迷走神经反应（包括头昏和头晕）、肌酸磷酸激酶一过性变化、注射部位红斑（皮肤发红）以及注射部位疼痛。 FDA 授予 Voceryq 突破性疗法认定和孤儿药认定。FDA 授予 Inovio Pharmaceuticals, Inc. 的 Voceryq 加速批准。 媒体： FDA 征求意见 202-690-6343 消费者： 888-INFO-FDA ### 模板文本 美国食品药品监督管理局（FDA）是美国卫生与公众服务部下属的一个机构，通过确保人用和兽用药物、疫苗及其他人用生物制品和医疗器械的安全性、有效性和保障性来保护公众健康。该机构还负责我国食品供应、化妆品、膳食补充剂、辐射发射电子产品以及烟草产品监管的安全与保障。 内容截至日期： 2026年10月30日 受监管产品 生物制品 关注FDA 在X上关注@US_FDA 在Facebook上关注FDA 在X上关注@FDAMedia
正文 · 英文原文
FDA News Release FDA Approves First Plasmid DNA-based Immunotherapy for Adults with Recurrent Respiratory Papillomatosis Groundbreaking therapy provides additional treatment option that fights the disease at its source, not only its symptoms More Press Announcements For Immediate Release: October 30, 2026 The U.S. Food and Drug Administration today granted accelerated approval for Voceryq (doruxapapogene ralaplasmid and lalikinogene sifuplasmid-gxfd) for treatment of adults with recurrent respiratory papillomatosis (RRP), a rare disease caused by human papillomavirus (HPV) that leads to the formation of small, wart-like growths in the patient’s airways. This approval, the first for a plasmid DNA-based immunotherapy in the U.S., offers patients a new therapeutic option that targets the root cause of RRP and has been shown to reduce the burden of surgical intervention, representing an important step forward for a patient population with limited treatment options. “Adults living with recurrent respiratory papillomatosis often face an exhausting and relentless cycle of repeated surgeries, managing their disease, but never truly escaping it. 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, B. Pharm., M.S., Director of the Center for Biologics Evaluation and Research (CBER). “For patients, that could mean fewer surgeries, fewer disruptions, and more time living their lives on their own terms. The FDA is proud to help bring that possibility within reach.” The agency based this accelerated approval on clinical trial data showing Voceryq helps reduce the frequency of surgical procedures required to remove growths in adult patients with RRP — an intermediate clinical endpoint reasonably likely to predict clinical benefit. The accelerated approval pathway allows the FDA to approve drugs or biologics for serious or life-threatening conditions based on an effect on a surrogate or intermediate clinical endpoint reasonably likely to predict clinical benefit. The manufacturer must conduct post-approval clinical trials to verify and describe Voceryq’s clinical benefit. RRP is a rare, chronic disease in which wart-like growths caused by HPV types 6 and/or 11 form in the airways, most often around the larynx (voice box), particularly the vocal cords. These growths, called papillomas, can cause symptoms like hoarseness, chronic cough, and voice changes, and in severe cases can spread to other parts of the respiratory tract, causing difficulty breathing. While surgery can remove the growths, there is no cure, and the virus persists in the tissue even after the growths are removed, so patients often need repeated procedures over many years. In some cases, RRP carries a risk of becoming cancerous over time. Voceryq is a recombinant plasmid DNA-based immunotherapy that delivers two plasmids (small pieces of DNA originally derived from bacteria) into the body’s cells, one of which contains the genetic instructions for specific HPV proteins and the other an immune response-enhancing molecule called IL-12. The therapy is designed to stimulate the immune system to respond to and attack cells infected by the specific types of HPV that cause RRP (types 6 and 11). Voceryq is administered via intramuscular (IM) injection using the CELLECTRA 5PSP Device, which delivers the dose and facilitates electroporation, a technique which uses brief electrical pulses to temporarily and reversibly create pores in the cell membranes to enable the delivery of Voceryq into the body’s cells. The safety and effectiveness of Voceryq was evaluated in an open-label multicenter clinical trial in 32 adults diagnosed with RRP. Eligible patients had two or more surgeries to remove laryngotracheal papillomas in the 12 months prior to Voceryq treatment, including a scheduled surgery at trial entry. Seven (27%) out of 26 patients who had at least three baseline surgeries did not require any surgery intervention during the first year after treatment. The most commonly reported adverse reactions to Voceryq were injection site reactions, headache, fatigue, hypertension (high blood pressure), nausea, and dizziness. Adverse effects associated with the electroporation technique may include brief muscle contractions or tetany, temporary bleeding at the administration site, anxiety, vasovagal reactions including lightheadedness and dizziness, transient changes in creatine phosphokinase, erythema (skin redness) at the injection site, and pain at the injection site. The FDA granted Voceryq Breakthrough and Orphan Drug designations. The FDA granted accelerated approval of Voceryq to Inovio Pharmaceuticals, Inc. Media: FDA Request for Comment 202-690-6343 Consumer: 888-INFO-FDA ### Boilerplate The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, radiation-emitting electronic products, and for regulating tobacco products. Content current as of: 10/30/2026 Regulated Product(s) Biologics Follow FDA Follow @US_FDA on X Follow FDA on Facebook Follow @FDAMedia on X