Regulatory & Policy

RemeGen adds 5th China indication for HER2 ADC, in urothelial carcinoma

IMPORTANT NOTICE: The source data describes a regulatory approval by China's NMPA — not a regulatory submission or filing. The article below accurately...

Edited by Robert McTiernan··3 min read

RemeGen Co., Ltd. announced that China's National Medical Products Administration (NMPA) has approved Aidixi (disitamab vedotin) in combination with toripalimab for the treatment of HER2-expressing locally advanced or metastatic urothelial carcinoma. The approval marks the fifth approved indication for disitamab vedotin in China and the drug's second approval in urothelial carcinoma, extending its reach from HER2-overexpressing disease to the broader HER2-expressing population.

Disitamab vedotin is an antibody-drug conjugate that targets the HER2 protein on tumour cells. It holds Breakthrough Therapy designations from both the US FDA and China's NMPA, and has now accumulated five approved indications in China: HER2-overexpressing locally advanced or metastatic gastric cancer; HER2-overexpressing locally advanced or metastatic urothelial carcinoma; HER2-positive advanced breast cancer with liver metastases; HER2-low expressing breast cancer with liver metastases; and the newly approved combination with toripalimab for HER2-expressing locally advanced or metastatic urothelial carcinoma. The asset is being developed in parallel for the US market, where it has attracted regulatory attention as a potential entrant in a competitive ADC landscape that includes enfortumab vedotin, which targets Nectin-4, and sacituzumab govitecan, a Trop-2-directed ADC, both approved in urothelial carcinoma.

The latest decision was based on data from the randomized, controlled, multicenter Phase III trial RC48-C016, which enrolled 484 subjects across 74 clinical trial centres in China. The study used dual primary endpoints of progression-free survival (PFS) and overall survival (OS), both of which were met with statistical significance as of the March 31, 2025 data cut. Median PFS reached 13.1 months in the disitamab vedotin plus toripalimab arm, compared with approximately half that in the platinum-based chemotherapy group, representing a 64% reduction in the risk of disease progression or death. Median OS was 31.5 months, nearly double that of the chemotherapy comparator, with a 46% reduction in the risk of death. The objective response rate was 76.1% overall, including 65.5% in the HER2 IHC 1+ subgroup, and the disease control rate reached 91.4%. Median duration of response was 14.6 months.

The RC48-C016 data were presented at the Presidential Symposium of the European Society for Medical Oncology Annual Congress in October 2025 and simultaneously published in the New England Journal of Medicine, giving the dataset broad international visibility before the domestic regulatory decision was issued.

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The trial's scope is clinically relevant in several respects. It enrolled patients across the full spectrum of HER2 expression — IHC 1+, 2+, and 3+ — and the PFS and OS benefits were consistent regardless of cisplatin eligibility or HER2 expression level. This breadth distinguishes the indication from earlier HER2-targeted approvals in urothelial carcinoma, which have generally been restricted to HER2-overexpressing subgroups. The grade ≥3 treatment-related adverse event rate was 55.1%, the company said. The result is also notable as the first Phase III trial to demonstrate, in a head-to-head comparison, that the combination of a HER2-targeting ADC and immunotherapy is superior to platinum-based chemotherapy as first-line treatment for HER2-expressing advanced urothelial carcinoma, as noted by Remegen.

Urothelial carcinoma is among the more common malignancies globally, with the highest incidence and mortality among male genitourinary tumours. Approximately 90% of cases originate in the bladder, with the remainder arising in the renal pelvis or ureter. A Frost & Sullivan analysis cited by RemeGen projects global new UC cases reaching 662,000 by 2030, of which approximately 106,000 are expected in China. Around 20% of patients present with metastatic or unresectable disease at diagnosis, and more than half of those eligible for first-line treatment are intolerant to platinum-based regimens — a gap that combination regimens pairing HER2-targeted agents with checkpoint inhibitors are increasingly being designed to address.

Of note, the approval is also relevant to Pfizer through its 2021 licensing agreement, via Seagen, for disitamab vedotin. Under the deal, Seagen—now part of Pfizer—obtained rights to develop and commercialize the drug outside Greater China, where RemeGen retains control. Pfizer is continuing global development of disitamab vedotin across multiple solid tumors, including breast and urothelial cancers, in studies conducted outside China.

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