FDA加速批准头颈癌新药Rybrevant Faspro：复发转移患者客观缓解率42%
美国食品药品监督管理局（FDA）11月5日授予 amivantamab and hyaluronidase-lpuj（商品名 Rybrevant Faspro，Janssen Biotech 公司）加速批准，用于治疗复发或转移性头颈部鳞状细胞癌（HNSCC）成人患者，适用人群为经铂类化疗和抗 PD-(L)1 抗体治疗后病情仍进展者。FDA 同时说明，该药不适用于 HPV 相关的口咽鳞状细胞癌患者。
疗效在 OrigAMI-4 试验（NCT06385080）中评估。这是一项多中心、开放标签、多队列临床试验，主要疗效人群包括 102 名复发或转移性头颈部鳞状细胞癌患者，不含经 p16 检测确认的 HPV 相关口咽癌患者。入组患者此前均接受过铂类化疗和抗 PD-(L)1 抗体治疗，按体重以推荐剂量每三周皮下注射该药单药治疗，直至病情进展或出现不可耐受的毒性。
主要疗效指标为客观缓解率与缓解持续时间，由盲态独立中心审查按 RECIST v1.1 标准判定。结果显示，客观缓解率为 42%（95% 置信区间：32% 至 52%），56% 的应答者缓解持续时间达到或超过 6 个月。
安全性方面，该药单药的处方信息列出的警告与注意事项包括超敏反应与给药相关反应、间质性肺病或肺炎、皮肤不良反应、眼部毒性以及胚胎-胎儿毒性。推荐剂量按患者体重确定，具体用法见处方信息，完整处方信息将刊登于 Drugs@FDA。
此次审评在 FDA 肿瘤学卓越中心的 Project Orbis 框架下进行，FDA 与巴西卫生监管机构 ANVISA、加拿大卫生部、以色列卫生部和新加坡 HSA 合作审评，相关申请在其他监管机构处可能仍在提交或审评过程中。审评使用了申请人自愿提交的 Assessment Aid，该申请获得优先审评资格，该药此前已获突破性疗法认定。
FDA Grants Accelerated Approval to Rybrevant Faspro for Recurrent or Metastatic Head and Neck Cancer, With a 42% Response Rate
The U.S. Food and Drug Administration (FDA) on November 5 granted accelerated approval to amivantamab and hyaluronidase-lpuj (Rybrevant Faspro, Janssen Biotech, Inc.) for adults with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) whose disease has progressed on or after platinum-based chemotherapy and an anti-PD-(L)1 antibody. The FDA said the drug is not indicated for patients with HPV-associated oropharyngeal squamous cell carcinoma.
Efficacy was evaluated in OrigAMI-4 (NCT06385080), a multicenter, open-label, multi-cohort clinical trial. The primary efficacy population included 102 patients with recurrent or metastatic HNSCC and did not include patients with HPV-associated oropharyngeal cancer based on p16 testing. Eligible patients had previously received platinum-based chemotherapy and an anti-PD-(L)1 antibody, and received single-agent, weight-based amivantamab and hyaluronidase-lpuj subcutaneously every three weeks at the recommended dose until disease progression or unacceptable toxicity.
The major efficacy outcome measures were overall response rate and duration of response, determined by blinded independent central review according to RECIST v1.1. The overall response rate was 42% (95% CI: 32%, 52%), and 56% of responders had a duration of response of six months or longer.
The prescribing information for the drug as a single agent includes warnings and precautions for hypersensitivity and administration-related reactions, interstitial lung disease and pneumonitis, dermatologic adverse reactions, ocular toxicity, and embryo-fetal toxicity. The recommended dose is based on body weight, and full prescribing information will be posted on Drugs@FDA.
The review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence, with the FDA collaborating with the Brazilian Health Regulatory Agency (ANVISA), Health Canada, the Israel Ministry of Health, and Singapore's HSA; the applications may still be undergoing submission or review at the other agencies. The review used the Assessment Aid, a voluntary submission from the applicant. The application was granted priority review, and the drug had received breakthrough therapy designation.
来源：美国食品药品监督管理局
发布：GSM·美国（发布链接）
图片来源：图：美国食品药品监督管理局（FDA）位于马里兰州 Silver Spring 的 White Oak 园区总部大楼。FDA 官方图，公共领域，经 Wikimedia Commons。 / Photo: FDA headquarters buildings at the White Oak campus in Silver Spring, Maryland. U.S. Food and Drug Administration, public domain, via Wikimedia Commons.
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