TOKYO, Japan and NEW YORK, U.S., April 20, 2026

Astellas Pharma Inc. and Pfizer Inc. have announced that the U.S. Food and Drug Administration (FDA) has granted Priority Review to their supplemental Biologics License Application (sBLA) for PADCEV™ (enfortumab vedotin) in combination with Keytruda® (pembrolizumab) as a perioperative treatment for muscle-invasive bladder cancer (MIBC) regardless of cisplatin eligibility. This milestone underscores the potential of the combination to become a new standard of care, addressing a major unmet need in patients with high recurrence risk following surgery.

FDA Priority Review Signals Transformative Oncology Potential

The FDA Priority Review designation is granted to therapies that may offer significant improvements over existing treatment options, highlighting the clinical importance of this combination therapy. The regulatory decision is based on data from the Phase 3 EV-304 (KEYNOTE-B15) trial, which demonstrated statistically significant improvements in event-free survival (EFS) and overall survival (OS) compared to standard chemotherapy.

Notably, the combination therapy achieved a 47% reduction in the risk of disease progression, recurrence, or death, along with a 35% reduction in the risk of death, when compared to gemcitabine plus cisplatin chemotherapy. Additionally, the regimen demonstrated a pathological complete response (pCR) rate of 55.8%, significantly higher than the 32.5% observed in the chemotherapy arm, reinforcing its clinical efficacy.

The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of August 17, 2026, marking a key regulatory milestone that could accelerate patient access to this innovative treatment.

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Expanding Treatment to All MIBC Patients Regardless of Eligibility

Currently, perioperative treatment options for muscle-invasive bladder cancer are limited, particularly for patients who are ineligible for cisplatin-based chemotherapy, which remains the standard of care. The new sBLA submission aims to expand the use of PADCEV plus Keytruda to all patients with MIBC, regardless of their eligibility for cisplatin therapy.

This expansion is highly significant, as approximately 50% of patients experience disease recurrence even after surgery, highlighting the urgent need for more effective therapies. The combination leverages the targeted action of an antibody-drug conjugate (ADC) with the immune activation of a PD-1 inhibitor, creating a synergistic anti-tumor effect that addresses multiple pathways in cancer progression.

The therapy is administered in both neoadjuvant (before surgery) and adjuvant (after surgery) settings, aiming to reduce tumor burden prior to surgery and prevent recurrence post-surgery, offering a comprehensive treatment approach.

Innovative Mechanism Combines ADC and Immunotherapy

PADCEV (enfortumab vedotin) is a first-in-class Nectin-4-directed antibody-drug conjugate (ADC) that delivers a potent cytotoxic agent directly to cancer cells, inducing cell cycle arrest and apoptosis. When combined with Keytruda (pembrolizumab), a PD-1 immune checkpoint inhibitor, the regimen enhances the body’s immune response against tumor cells.

The safety profile observed in the Phase 3 study was consistent with previously established data, with no new safety signals identified, supporting its continued development and potential regulatory approval. However, known risks such as skin reactions, peripheral neuropathy, and metabolic effects require careful monitoring under clinical practice guidelines.

This combination represents a next-generation oncology strategy, integrating targeted therapy and immunotherapy to improve outcomes in a disease area with historically limited progress.

The FDA’s Priority Review designation for PADCEV plus Keytruda marks a significant advancement in the treatment of muscle-invasive bladder cancer, with the potential to transform the perioperative treatment landscape. Backed by strong Phase 3 clinical data, this combination therapy offers improved survival outcomes, broader patient eligibility, and a novel mechanism of action, positioning it as a promising new standard of care. As regulatory review progresses toward the August 2026 decision date, this development highlights the continued evolution of precision oncology and combination therapeutics in addressing critical unmet medical needs.

Source: Astellas, Pfizer press release