Posted by Michael Wonder on 17 Jan 2023
Orphagen Pharmaceuticals receives rare paediatric disease designation from FDA for OR-449 for the treatment of paediatric adrenocortical carcinoma
17 January 2023 - Orphagen Pharmaceuticals today announced that the US FDA has granted rare paediatric disease designation for OR-449 for the treatment of paediatric adrenocortical carcinoma.
OR-449 is a selective, first in class, potent and orally bioavailable small molecule antagonist to steroidogenic factor-1 (SF-1 or NR5A1), an orphan nuclear receptor and transcription factor that is essential for the growth and development of the adrenal gland.
