The FDA approved Pfizer and Arvinas' oral drug Veppanu for adults with ESR1‑mutant, ER‑positive, HER2‑negative advanced breast cancer after progression on prior hormone therapy. A phase 3 trial of 624 patients showed improved progression‑free survival versus fulvestrant and higher tumor shrinkage rates. The label warns of heart rhythm effects and potential fetal harm; the recommended dose is 200 mg once daily with food. A companion blood test, Guardant360 CDx, was also authorized to identify eligible patients.
FDA Clears Veppanu: New Oral Option for ESR1‑Mutant Advanced Breast Cancer
A Pfizer logo is displayed at a research facility in the La Jolla neighborhood of San Diego, California, U.S., September 30, 2025. REUTERS/Mike Blake
The U.S. Food and Drug Administration has approved Veppanu, an oral breast cancer therapy developed by Pfizer and Arvinas, for adults with a specific form of advanced disease. The drug is authorized for patients with estrogen receptor (ER)-positive, HER2-negative breast cancer that has metastasized or cannot be removed surgically, provided their tumors carry an ESR1 mutation and they have progressed on at least one prior hormone therapy.
Clinical Evidence and Benefits
The approval was based on a randomized late-stage trial of 624 participants that showed Veppanu significantly prolonged the time patients lived without disease worsening (progression-free survival) versus the established injectable hormone therapy fulvestrant. The FDA also noted that patients receiving Veppanu experienced tumor shrinkage at higher rates than those given the injectable comparator.
Safety, Dose, and Testing
The pill’s label warns of potential effects on heart rhythm and the possibility of harm to an unborn child, underscoring the need for cardiac monitoring and contraception counseling where appropriate. The recommended dose is 200 mg once daily with food, continued until disease progression or unacceptable toxicity.
The FDA simultaneously approved a companion blood-based diagnostic, Guardant360 CDx, to identify patients whose tumors carry the ESR1 mutation and who are therefore eligible for Veppanu treatment.
Industry Reaction and Next Steps
'Patients really are in need of a new modality, a new technology and a new way of hitting disease,' said Arvinas CEO Randy Teel, highlighting the need for additional options in stage IV disease.
Wedbush analyst Robert Driscoll described Veppanu’s efficacy as competitive with other approved therapies, while noting cross-trial differences complicate direct comparisons. Arvinas has indicated it expects to announce a commercialization agreement in the coming weeks, which should clarify availability and pricing.
Reporting: Padmanabhan Ananthan and colleagues; Editing: staff editors.
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