Posted by Michael Wonder on 14 Jan 2024
Drug Farm receives rare paediatric disease designation from the US FDA for DF-003 to treat ROSAH syndrome
13 January 2024 - Drug Farm announced that the US FDA has granted rare paediatric disease designation for the company’s alpha-kinase 1 inhibitor, DF-003, to treat patients with ROSAH (retinal dystrophy, optic nerve oedema, splenomegaly, anhidrosis and headache) syndrome.
DF-003 is currently being evaluated in a Phase 1 trial to assess safety and pharmacokinetics in normal healthy volunteers.
