SpringWorks Therapeutics receives breakthrough therapy designation for nirogacestat for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumours

SpringWorks Therapeutics

29 August 2019 - SpringWorks Therapeutics today announced the U.S. 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 tumours or deep fibromatosis. 

The breakthrough therapy designation was based on Phase 1 and Phase 2 data evaluating nirogacestat as a monotherapy in patients with desmoid tumors.

Previously, the FDA had granted nirogacestat orphan drug designation for the treatment of desmoid tumors (June 2018) and fast track designation for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumours or deep fibromatosis (November 2018).

Read SpringWorks Therapeutics press release

Michael Wonder

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Michael Wonder