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February 19, 2020
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FDA grants breakthrough therapy designation to Padcev regimen for advanced bladder cancer

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The FDA granted breakthrough therapy designation to enfortumab vedotin-ejfv in combination with pembrolizumab for the treatment of patients with unresectable locally advanced or metastatic urothelial cancer who are ineligible for first-line cisplatin-based chemotherapy, according to a press release from the agent’s manufacturer.

Enfortumab vedotin-ejfv (Padcev; Astellas Pharma, Seattle Genetics) is an antibody-drug conjugate directed against the nectin-4 protein on the surface of cancer cells.

The FDA granted accelerated approval to enfortumab vedotin-ejfv in December for treatment of adults with locally advanced or metastatic urothelial cancer who previously received a PD-1/PD-L1 inhibitor and a platinum-containing chemotherapy before or after surgery, as previously reported by Healio.

The FDA granted the breakthrough therapy designation based on results of the dose-escalation cohort and expansion cohort A of the phase 1b/phase 2 EV-103 trial, in which patients with advanced or metastatic urothelial cancer who were ineligible for cisplatin-based chemotherapy received the first-line combination of enfortumab vedotin-ejfv plus pembrolizumab (Keytruda, Merck).

Updated results of the study — presented at Genitourinary Cancers Symposium — showed an objective response rate of 73.3% for the combination therapy, including a 15.6% complete response rate and 93.3% disease control rate.

The most common treatment-related adverse events included fatigue, experienced by 58% of patients (11% grade 3), alopecia (53%) and peripheral sensory neuropathy (53%; 4% grade 3).

“The FDA’s breakthrough therapy designation reflects the encouraging preliminary evidence for the combination of Padcev and pembrolizumab in previously untreated advanced urothelial cancer to benefit patients who are in need of effective treatment options,” Andrew Krivoshik, MD, PhD, senior vice president and head of oncology therapeutics at Astellas, said in the press release. “We look forward to continuing our work with the FDA as we progress our clinical development program as quickly as possible.”

The FDA's breakthrough therapy designation helps expedite the development and review of drugs for serious or life-threatening conditions. The designation requires preliminary clinical evidence that the treatment may substantially improve upon at least one clinically significant endpoint compared with available therapies.

“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,” Roger Dansey, MD, chief medical officer for Seattle Genetics, said in the press release. “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.”