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Home » European Commission Grants SpringWorks’ Nirogacestat Orphan Status
European Commission Grants SpringWorks’ Nirogacestat Orphan Status
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October 4, 2019
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.
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