China's NMPA Approves PADCEV™ for Advanced Urothelial Cancer

Astellas Pharma Inc. has announced that China's National Medical Products Administration (NMPA) has approved enfortumab vedotin for treating adult patients with locally advanced or metastatic urothelial cancer (la/mUC) following platinum-based chemotherapy and PD-1/PD-L1 inhibitor treatment. This approval offers a new therapeutic option for Chinese patients grappling with this aggressive cancer, which affects both the upper and lower urinary tracts, including the bladder and urethra.

Urothelial cancer is known for its high morbidity and mortality rates. In 2022, over 92,000 people were diagnosed with bladder cancer in China, leading to approximately 41,000 deaths. The prognosis for patients with advanced or metastatic forms of the disease is particularly poor, underscoring the urgent need for new treatment modalities that can prolong survival and improve quality of life.

The approval of enfortumab vedotin is based on the results of two pivotal clinical trials: the global EV-301 trial and the China-specific EV-203 trial.

EV-301 was a global Phase 3 randomized trial that compared enfortumab vedotin to standard chemotherapy in patients with la/mUC who had previously received PD-1/PD-L1 inhibitors and platinum-based chemotherapy. The trial demonstrated a significant improvement in overall survival for patients treated with enfortumab vedotin, with a median overall survival of 12.88 months compared to 8.97 months for those on chemotherapy. Additionally, progression-free survival was better in the enfortumab vedotin group, further validating the efficacy of this treatment.

The China-specific EV-203 trial served as a bridging study to complement the global EV-301 data. EV-203 was a Phase 2, single-arm, open-label multicenter trial involving 40 Chinese patients with la/mUC. These patients had also received PD-1/PD-L1 inhibitors and platinum-based chemotherapy before participating in the trial. The results were encouraging, with an objective response rate of 37.5%, which includes both complete and partial responses. The safety profile of enfortumab vedotin was consistent with global data, showing that the majority of adverse events were manageable and mostly of grade 1–2 severity.

Medical experts in China have expressed optimism about the approval. Professor Guo Jun, who was the principal investigator of the EV-203 trial, noted that this milestone event would allow more la/mUC patients in China to access this innovative antibody-drug conjugate (ADC) treatment. Professor Dingwei Ye of Fudan University-Affiliated Cancer Hospital highlighted the benefits that enfortumab vedotin would bring to Chinese patients, particularly those who have exhausted other treatment options. Similarly, Professor Zhisong He emphasized that the approval of enfortumab vedotin expands the therapeutic arsenal available to oncologists.

Astellas Pharma’s Senior Vice President and Head of Oncology Development, Ahsan Arozullah, reiterated the company's commitment to driving scientific advancements in cancer treatment. He emphasized that the approval by the NMPA provides renewed hope for better patient outcomes in China, reinforcing Astellas' dedication to improving global health.

This approval has already been factored into Astellas' financial forecasts for the fiscal year ending March 31, 2025. The company, in partnership with Pfizer, is also conducting further trials to explore additional applications of enfortumab vedotin in various stages and types of urothelial cancer.

Overall, the approval of enfortumab vedotin marks a significant advancement in the treatment landscape for locally advanced or metastatic urothelial cancer in China, providing patients and healthcare providers with a promising new option in the fight against this challenging disease.