SpringWorks' oral, selective, small molecule, gamma-secretase inhibitor nirogacestat has been designated an orphan drug for the treatment of soft tissue sarcoma.

The firm is currently enrolling patients in the Phase III DeFi trial assessing nirogacestat for the treatment of adults with progressing desmoid tumours, which are a type of soft-tissue tumours 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 even death in the worst cases.

“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 US by the FDA,” said Saqib Islam, SpringWorks' chief executive.

“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.”