FDA Grants Breakthrough Therapy Designation for Treatment of Locally Advanced, Metastatic Urothelial Cancer


Enfortumab vedotin-ejfv in combination with the anti-PD1-therapy pembrolizumab for the treatment of patients with unresectable locally advanced or metastatic urothelial cancer who are unable to receive cisplatin-based chemotherapy in the first-line setting.

Officials with the FDA have granted Breakthrough Therapy designation for enfortumab vedotin-ejfv (Padcev, Seattle Genetics and Astellas Pharma) in combination with the anti-PD1-therapy pembrolizumab (Keytruda, Merck) for the treatment of patients with unresectable locally advanced or metastatic urothelial cancer that are unable to receive cisplatin-based chemotherapy in the first-line setting.1

Enfortumab vedotin-ejfv is a combination of 2 difference types of drugs, called an antibody-drug conjugate. The antibody part acts like a homing signal, bringing the chemo drug, monomethyl auristantin E, directly to bladder cancer cells with a protein called Nectin-4 on them and killing them, according to the American Cancer Society.2

Enfortumab vedotin-ejfv was approved by the FDA in December 2019 for the treatment of adult patients with locally advanced or metastatic urothelial cancer who have previously received a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor and a platinum-containing chemotherapy before or after surgery or in a locally advanced or metastatic setting. The drug was approved for this indication under the agency’s Accelerated Approval Program based on tumor response rate.1

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Metastatic urothelial cancer is an aggressive and devastating disease with limited treatment options, according to Jonathan E. Rosenberg, MD, medical oncologist, chief, Genitourinary Medical Oncology Service, Memorial Sloan Kettering Cancer Center in New York. Enfortumab vedotin-ejfv is a significant advancement for patients that previously had limited options after other therapies failed, he added.3

The drug’s new Breakthrough Therapy designation was granted based on results from the dose-escalation cohort and expansion cohort A of the phase 1b/2 trial, EV-103, which evaluated patients with locally advanced or metastatic urothelial cancer that were unable to receive cisplatin-based chemotherapy treated in the first-line setting with enfortumab vedotin-ejfv in combination with pembrolizumab.1

“This is an important step in our investigation of PADCEV in combination with pembrolizumab as a first-line therapy for patients with advanced urothelial cancer who are unable to receive cisplatin-based chemotherapy,” said Roger Dansey, MD, Chief Medical Officer, Seattle Genetics, in a prepared statement.1 “Based on encouraging early clinical activity, we recently initiated a phase 3 trial of this platinum-free combination and look forward to potentially addressing an unmet need for patients.”

In this trial, 12% of participants had a complete response, meaning that their tumors were no longer detectable, and 32% had a partial response, meaning their tumors got smaller but did not disappear, according to the American Cancer Society.2

EV-103 is an ongoing, multi-cohort, open-label, multicenter phase 1b/2 trial of PADCEV alone or in combination, evaluating safety, tolerability and efficacy in muscle invasive, locally advanced and first- and second-line metastatic urothelial cancer.1


  • Seattle Genetics and Astellas Receive FDA Breakthrough Therapy Designation for PADCEV™ (enfortumab vedotin-ejfv) in Combination with Pembrolizumab in First-Line Advanced Bladder Cancer [news release]. Seattle Genetics website. Published February 19, 2020. https://investor.seattlegenetics.com/press-releases/news-details/2020/Seattle-Genetics-and-Astellas-Receive-FDA-Breakthrough-Therapy-Designation-for-PADCEV-enfortumab-vedotin-ejfv-in-Combination-with-Pembrolizumab-in-First-Line-Advanced-Bladder-Cancer/default.aspx. Accessed February 19, 2020.
  • FDA Approves Padcev (Enfortumab vedotin-ejfv) for Bladder Cancer. American Cancer Society website. Published December 20, 2019. http://www.cancer.org/latest-news/fda-approves-padcev-enfortumab-vedotin-ejfv-for-bladder-cancer.html. Accessed February 19, 2020.
  • Nessel J. Enfortumab Vedotin Receives FDA Approval for Locally Advanced or Metastatic Urothelial Cancer. Pharmacy Times website. https://www.pharmacytimes.com/news/enfortumab-vedotin-receives-fda-approval-for-locally-advanced-or-metastatic-urothelial-cancer Published December 19, 2019. Accessed February 20, 2020.

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