Rare Diseases

European Commission Grants Orphan Drug Designation for Nirogacestat for the Treatment of Soft Tissue Sarcoma. Nirogacestat is a selective oral small molecule, gamma-secretase inhibitor under development by SpringWork Therapeutics. Currently, in Phase III development, the drug is investigated for the treatment of progressive Desmoid tumors, which are a type of soft-tissue tumour. The drug has already been recommended by the FDA in June 2018. Desmoid tumors are an abnormal increase in the connective tissues. The connective tissues are responsible for the strength and flexibility of the bones, ligaments, and muscles. These tumors are considered locally invasi...