SpringWorks Therapeutics has launched its experimental drug, nirogacestat for the treatment of adult patients with rare and progressive cancer known as desmoid tumors.
Nirogacestat is an oral, selective, small molecule, a gamma-secretase inhibitor that works by blocking proteolytic activation of Notch receptors. Notch signaling plays a vital role in desmoid tumor growth.
The breakthrough designation given by the US Food and Drug Administration was based on data obtained from phase 1 and phase 2 clinical trials that evaluated nirogacestat as monotherapy in 24 patients with desmoid tumors.
The results demonstrated a 100% disease control rate as measured by the RECIST criteria.
Nirogacestat was generally well-tolerated with the most common adverse events being diarrhea, skin disorders, and hypophosphatemia.
Currently, the phase 3 DeFi trial is investigating nirogacestat in adult patients with desmoid tumors.
Desmoid tumors, also known as aggressive fibromatosis or desmoid-type fibromatosis, are generally non-malignant, soft tissue tumors that are abnormal growths of connective tissue.
The tumors are often debilitating and disfiguring with symptoms that vary greatly depending on the size and location.
However, they generally occur in the upper and lower extremities, abdominal walls, thoracic areas and the head and neck.
It causes severe pain, internal bleeding, limits a range of motion and may turn fatal in rare cases.
The cause of desmoid tumors is unknown, and there are no FDA-approved treatments currently available.