Briefs · Frontier Science
Veppanu Becomes the First Approved PROTAC
For twenty-five years, PROTACs were a clever idea. On 1 May, they became a medicine.
The FDA approved Veppanu, known as vepdegestrant, for adults with ER-positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer. The patients had already progressed on at least one line of endocrine therapy.
Veppanu is not a blocker, but rather a binman. It does not sit on the oestrogen receptor and hope for the best. It hauls the receptor to the cell’s own disposal machinery and has it destroyed.
The evidence came from the VERITAC-2 trial. In 270 patients with ESR1 mutations, median progression-free survival was 5.0 months on Veppanu, against 2.1 months on fulvestrant. The risk of progression or death fell by 43 percent.
The drug was developed by Arvinas with Pfizer. It is taken as a 200 mg tablet once daily. The FDA also approved a companion test to find the ESR1 mutation.
A quarter-century of chemistry has produced a deliberate demolition of a disease-driving protein. The undruggable just got a demolition date.
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