FDA Grants Approval to First Treatment for Uncommon Non-Malignant Tumors

December 05, 2023

The U.S. Food and Drug Administration has granted approval for Ogsiveo (nirogacestat) tablets, marking a groundbreaking development for adult patients with advancing desmoid tumors requiring systemic treatment. 

This approval positions Ogsiveo as the first-ever drug endorsed for treating desmoid tumors, a rare subtype of non-cancerous soft tissue sarcomas. Desmoid tumors, while non-cancerous, can exhibit local aggressiveness, causing pain, restricted mobility, and diminished quality of life as they invade nearby structures and organs. Surgical removal, historically the preferred treatment, presents risks of recurrence or additional health complications. 

Ogsiveo's efficacy was validated in an international, multicenter, randomized, double-blind, placebo-controlled trial involving 142 adult patients with progressing desmoid tumors deemed unsuitable for surgery. The trial demonstrated Ogsiveo's significant improvement in progression-free survival and objective response rate compared to the placebo, offering a much-needed therapeutic option for patients facing limited alternatives.

SOURCE: https://www.fda.gov/news-events/press-announcements/fda-approves-first-therapy-rare-type-non-cancerous-tumors