Desmoid Tumors and the Approval of Ogsiveo

Описание: Bernd Kasper, MD, PhD, Mannheim Cancer Center at University of Heidelberg in Germany, and Principal Investigator of the DeFi trial, discusses desmoid tumors and the approval of Ogsiveo (nirogacestat).

A desmoid tumor is a benign, locally invasive soft tissue tumor. It is associated with a high recurrence rate but has no metastatic potential. These tumors only affect about five in one million per year. They are characterized by their variable presentation and unpredictable behavior.

As Dr. Kasper explains, desmoid tumors are traditionally treated in the same manner as malignant sarcoma, through surgeries and chemotherapies. However, advancements in treatment have allowed a shift toward therapies specifically designed to treat this designation.

Nirogacestat, a gamma secretase inhibitor, makes history as the first approved drug for desmoid tumors specifically. The Phase 3 DeFi trial was an international, double-blind, randomized, placebo-controlled trial of nirogacestat in adults with progressing desmoid tumors. Nirogacestat met the primary endpoint with a 71% reduction in the risk of disease progression. Side effects were common but of low grade.

The advancement also gives hope to patients in terms of insurance coverage and reimbursement for the treatment.

For more information on desmoid tumors and other musculoskeletal disorders, visit https://checkrare.com/diseases/muscul...