European Commission Grants SpringWorks’ Nirogacestat Orphan Status

The European Commission granted orphan drug designation to SpringWorks Therapeutics for nirogacestat to treat soft tissue sarcoma.

Nirogacestat is an oral, selective, small molecule, gamma-secretase inhibitor that is currently in phase 3 clinical development to treat desmoid tumor or soft tissue sarcoma.

The drug has been investigated in 24 patients with desmoid tumors in phase 1 and phase 2  trials. The treatment demonstrated a 100 percent disease control rate.