Arvinas, Inc., in partnership with Pfizer Inc., has received acceptance from the U.S. Food and Drug Administration (FDA) for a New Drug Application (NDA) for its drug vepdegestrant. This investigational oral therapy is intended for the treatment of patients with a specific type of advanced or metastatic breast cancer: estrogen receptor-positive (ER+), human epidermal growth factor receptor 2-negative (HER2-), and ESR1-mutated disease. The acceptance of the NDA is a significant step toward making this potential new treatment available to patients. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) action date of June 5, 2026, by which it is expected to complete its review.
Vepdegestrant is a first-of-its-kind drug, utilizing a unique mechanism known as PROteolysis TArgeting Chimera (PROTAC). This technology harnesses the body's natural protein degradation system to specifically destroy disease-causing proteins. Vepdegestrant is the first PROTAC to show a clinical benefit in patients with breast cancer, marking a notable milestone in cancer therapy. The drug is being developed as a potential monotherapy for patients who have already received other endocrine-based therapies.
The NDA submission is supported by compelling data from the global, randomized Phase 3 VERITAC-2 clinical trial. This study compared vepdegestrant to fulvestrant, the current standard of care. The trial data, recently presented at the American Society of Clinical Oncology (ASCO) 2025 Annual Meeting and published in The New England Journal of Medicine, demonstrated that vepdegestrant provided a statistically significant and clinically meaningful improvement in median progression-free survival. The VERITAC-2 trial enrolled 624 patients across 25 countries, 43% of whom had ESR1 mutations, confirming the drug's potential efficacy in this specific patient population.
Dr. John Houston, Chairperson, CEO, and President at Arvinas, highlighted the importance of this development, noting that many patients in this advanced stage of breast cancer have limited treatment options. The company believes that vepdegestrant, with its demonstrated efficacy and favorable tolerability, could become a best-in-class treatment option. Arvinas and Pfizer are committed to working closely with the FDA to secure approval and make this treatment available as quickly as possible. The FDA has also granted vepdegestrant Fast Track designation, underscoring the high unmet medical need and the drug's potential to offer a meaningful new option for patients. This collaboration between Arvinas and Pfizer involves a shared commitment to worldwide development costs, commercialization expenses, and profits.