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The European Medicines Agency has validated a marketing authorization application for varegacestat, an oral gamma-secretase inhibitor, in adults with desmoid tumors. The application is backed by the phase 3 RINGSIDE trial, where varegacestat slashed the risk of disease progression or death by 84% vs. placebo. The FDA has also accepted a new drug application, with a target action date of April 28, 2027.
The European Medicines Agency (EMA) has validated a marketing authorization application (MAA) for varegacestat — an investigational oral gamma-secretase inhibitor — in adult patients with desmoid tumors, kicking off the agency's formal centralized review. The drug works by blocking gamma-secretase to disrupt Notch signaling, a key driver of desmoid tumor biology alongside the Wnt pathway.
The application is anchored by the phase 3 RINGSIDE trial, a randomized, double-blind, placebo-controlled study in 156 patients with progressing desmoid tumors. Varegacestat delivered striking efficacy results, and a separate FDA review is also underway in the U.S., with a target action date of April 28, 2027.
By the Numbers:
Why it matters: Desmoid tumors are rare, locally aggressive soft tissue tumors with no approved systemic therapies in Europe. Varegacestat's robust efficacy and pain reduction data could offer patients a meaningful new treatment option on both sides of the Atlantic.
