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 tumor that grows from connective tissue. It is associated with a high recurrence rate but has no metastatic potential. These tumors can be found anywhere in the body but commonly occur in the abdomen, shoulders, upper arms, and thighs. The speed of growth also varies by patient.
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, is the first approved drug for desmoid tumors. In the Phase 3 DeFi trial, nirogacestat met the primary endpoint with a 71% reduction in the risk of disease progression, measured by how much the tumor grew, showcasing impressive efficacy. 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/musculoskeletal-diseases/
