The European Medicines Agency has validated Immunome’s marketing authorisation application for varegacestat, an investigational oral, once daily gamma secretase inhibitor for adults with desmoid tumours, the company announced on October 30. Validation confirms the application is complete and starts the review under the European Union centralised procedure. It is not an approval, and the company gave no date for a European decision.
The application is supported by RINGSIDE, a global, randomized, double blind, placebo controlled Phase 3 trial in 156 patients with progressing desmoid tumours, which the company describes as the largest randomized study in this population. Varegacestat met its primary endpoint of progression free survival, with an 84 percent reduction in the risk of disease progression or death against placebo (hazard ratio 0.16, p less than 0.0001). The objective response rate was 56 percent against 9 percent on placebo, and an exploratory analysis showed a median best change in tumour volume of minus 83 percent against plus 11 percent on placebo.
The safety profile was consistent with the gamma secretase inhibitor class. The most common adverse events on treatment were diarrhea at 82 percent, fatigue at 44 percent, rash at 43 percent, nausea at 35 percent and cough at 34 percent, and 95 percent of events were grade 1 or 2.
In the United States, the Food and Drug Administration accepted Immunome’s new drug application for the same indication in July 2026 and set a PDUFA target action date of April 28, 2027. Desmoid tumours are non-metastatic soft tissue tumours that recur readily and can cause severe pain and disability; the company estimates 1,000 to 1,650 new diagnoses a year in the United States and 10,000 to 11,000 actively managed patients.