The FDA has cleared an investigational new drug application for an alpha-kinase 1 inhibitor for clinical evaluation in those with retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis and headache, or ROSAH, syndrome.
Drug Farm, manufacturer of DF-003, said in a press release that the first-in-class oral therapeutic will be examined in a pending clinical trial to examine safety, efficacy and pharmacokinetics in this patient population.
DF-003 is a highly selective inhibitor of the gain-of-function ALPK1 mutation responsible for causing ROSAH syndrome, whose results in a completed phase