SpringWorks Therapeutics announced the European Commission has granted Orphan Drug Designation for nirogacestat, an oral, selective, small molecule, gamma-secretase inhibitor, for the treatment of soft tissue sarcoma.
SpringWorks is currently enrolling patients in the Phase 3 DeFi trial of nirogacestat for the treatment of adult patients with progressing desmoid tumors, which are a type of soft-tissue tumors that are often treated by sarcoma specialists. Desmoid tumors are rare and often debilitating and disfiguring, and 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.1 It is estimated that 1,000 to 1,500 new desmoid tumor patients are diagnosed each year in the United States.2,3 There are currently no therapies approved by the U.S. Food and Drug Administration (FDA) for the treatment of desmoid tumors.
“This Orphan Drug Designation in the European Union is another important development for SpringWorks and follows the Orphan Drug, Fast Track and Breakthrough Therapy Designations already granted for nirogacestat in the U.S. by the FDA,” said Saqib Islam, Chief Executive Officer of SpringWorks. “We are currently enrolling adult patients in our DeFi trial and will continue to work closely with global regulators with the goal of bringing nirogacestat to patients as quickly as possible.”
The European Commission grants orphan medicinal status for products intended for the treatment, prevention or diagnosis of life-threatening or very serious conditions that affect no more than 5 in 10,000 people in the European Union, and where the product represents a significant benefit over existing treatments. Orphan designation provides companies with certain benefits and incentives in the EU, including a 10-year period of market exclusivity after product approval, reduced regulatory fees and protocol assistance.4
Nirogacestat previously received Orphan Drug Designation from the FDA for the treatment of desmoid tumors (June 2018), and Fast Track and Breakthrough Therapy Designations from the FDA for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis (November 2018 and August 2019).