Less pain, more movement: a daily pill for desmoid tumours proves itself on the measures patients actually feel

Desmoid tumours are not cancer — they do not spread — but they grow aggressively into muscle and organs, and for the roughly 1,000 to 1,650 people diagnosed each year in the USA they can mean debilitating pain, deformity and lost movement. Shrinking the tumour is only half the point; feeling better is the other half. At the ESMO congress in Madrid, researchers shared the patient-reported results from RINGSIDE, the largest randomised trial ever run in this condition, with 156 patients taking the once-daily pill varegacestat or a placebo. By week 48, patients on the pill reported a clear fall in symptom burden while the placebo group's worsened, and their physical function improved as the placebo group's declined. The results build on the trial's headline findings: tumours shrank in 56% of patients on varegacestat against 9% on placebo, and the risk of the disease progressing fell by 84%. The US Food and Drug Administration accepted the company's approval application in July 2026, with a decision due by April 28, 2027, and a European submission is planned by the end of this year.

SDG 3 · Good health and well-being — Mechanism: tech innovation