Breakthrough Drug Launched to Treat Desmoid Tumors in Adult Patients
SpringWorks Therapeutics has introduced an experimental drug called nirogacestat for the treatment of rare and progressive cancer called desmoid tumors in adult patients.
Nirogacestat is an oral, selective, small molecule that acts as a gamma-secretase inhibitor, specifically targeting the proteolytic activation of Notch receptors. Notch signaling plays a crucial role in the growth of desmoid tumors.
The drug received breakthrough designation from the US Food and Drug Administration (FDA) based on data obtained from phase 1 and phase 2 clinical trials involving 24 patients with desmoid tumors who were treated with nirogacestat as monotherapy. The results showed a 100% disease control rate according to the RECIST criteria.
Nirogacestat demonstrated favorable tolerability, with the most commonly reported adverse events being diarrhea, skin disorders, and hypophosphatemia.
Currently, the phase 3 DeFi trial is underway, investigating the efficacy of nirogacestat in adult patients with desmoid tumors.
Desmoid tumors, also known as aggressive fibromatosis or desmoid-type fibromatosis, are typically non-malignant soft tissue tumors characterized by abnormal growths of connective tissue. These tumors can cause significant debilitation and disfigurement, with symptoms varying depending on their size and location. Desmoid tumors commonly occur in the upper and lower extremities, abdominal walls, thoracic areas, as well as the head and neck. They often result in severe pain, internal bleeding, limited range of motion, and in rare cases, may even be fatal.
The cause of desmoid tumors is unknown, and there are no FDA-approved treatments currently available.
