SpringWorks Therapeutics reports that the FDA has granted Breakthrough Therapy Designation for nirogacestat, an oral, selective, small molecule, gamma-secretase inhibitor, for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis.
Desmoid tumors are rare and often debilitating and disfiguring soft-tissue tumors that can aggressively invade surrounding healthy tissues and cause significant morbidities, including severe pain, internal bleeding, incapacitating loss of range of motion, and, in rare cases, death. It is estimated that 1,000 to 1,500 new desmoid tumor patients are diagnosed each year in the United States. There are currently no therapies approved by the FDA for the treatment of desmoid tumors. more