The U.S. Food and Drug Administration (FDA) recently approved vepdegestrant (marketed as Veppanu) for adults with estrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2)-negative breast cancer and mutations in the ESR1 gene. The therapy was developed by pharmaceutical companies Arvinas and Pfizer.
Eligible patients must have advanced or metastatic breast cancer and experience disease progression after receiving endocrine therapy.
Vepdegestrant works by targeting and destroying estrogen receptors to slow cancer growth. It belongs to a novel class of drugs known as proteolysis targeting chimeras (PROTACs) and is the first therapy of its kind to receive FDA approval.
“This first approval will encourage drug developers to explore the use of PROTACs for other diseases such as neurodegeneration (dementia) and other challenging diseases,” said Craig Crews, a Yale chemist who led research efforts into the development of PROTACs.
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Alongside vepdegestrant, the FDA approved Guardant360 CDx to identify patients with ESR1 mutations who may be eligible for treatment. Guardant360 CDx is currently approved for the detection of numerous tumor mutations.
Approval of vepdegestrant was based on findings from the VERITAC-2 clinical trial. The study randomized 624 individuals with ER-positive, HER2-negative breast cancer to receive oral vepdegestrant or intramuscular fulvestrant. Of the participants, 270 patients harbored ESR1 mutations.
Among those with ESR1 mutations, participants receiving vepdegestrant had a median progression-free survival of 5 months, compared with 2.1 months among those receiving fulvestrant. Moreover, the objective response rate was 19% in the vepdegestrant arm and 4% in the fulvestrant arm.
Individuals are recommended to take 200 mg vepdegestrant orally once per day with food. Patients should continue treatment unless progression or side effect occur. The drug carries warnings for changes in heart rhythm and fetal toxicity.
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