The U.S. Food and Drug Administration has granted Priority Review to a supplemental Biologics License Application (sBLA) filed by Astellas Pharma Inc. and Pfizer Inc., seeking to expand the use of their cancer therapy combination for patients with muscle-invasive bladder cancer (MIBC). The application centers on the use of PADCEV in combination with Keytruda or its subcutaneous formulation Keytruda QLEX.
The combination therapy was initially approved in November 2025 for perioperative use—meaning before and after surgery—in patients with MIBC who are ineligible for cisplatin-based chemotherapy. The newly submitted sBLA aims to broaden this indication to include all patients with MIBC, regardless of their eligibility for cisplatin treatment, potentially expanding access to a wider patient population.
The FDA’s Priority Review designation is reserved for therapies that may offer significant improvements over existing treatments or address unmet medical needs. Under the Prescription Drug User Fee Act (PDUFA), the agency has set a target decision date of August 17, 2026.
The submission is supported by results from the Phase 3 EV-304 clinical trial, also known as KEYNOTE-B15. Data from the study, presented at the ASCO Genitourinary Cancers Symposium, demonstrated promising outcomes for the combination regimen. Patients receiving perioperative enfortumab vedotin plus pembrolizumab experienced a 47% reduction in the risk of disease recurrence, progression, or death compared to standard chemotherapy. Additionally, the treatment reduced the risk of death by 35%.
Notably, the study also reported a higher pathological complete response (pCR) rate—55.8% in the combination therapy group versus 32.5% in patients treated with standard gemcitabine and cisplatin chemotherapy—indicating a stronger tumor response prior to surgery.
Experts from both companies emphasized the potential impact of the therapy. They highlighted that recurrence rates in MIBC remain high despite surgery, underscoring the need for more effective perioperative treatment options. If approved, the combination could significantly improve long-term outcomes and reduce recurrence risks for a broader range of patients.
The safety profile observed in the EV-304 trial was consistent with previous findings, with no new safety concerns identified. Astellas and Pfizer plan to engage with global regulatory authorities to pursue further approvals based on these results, signaling a potential shift in the treatment landscape for bladder cancer.