FDA clears investigational new drug application to alpha kinase inhibitor for ROSAH syndrome

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 1 study in healthy participants supported a once-a-day oral dosing regimen in the upcoming ROSAH patient trial.

The FDA granted the drug rare pediatric disease designation in January.

“We have developed a precision drug that inhibits mutant ALPK1 and may stop progression of disease in patients afflicted with ROSAH,” Jeysen Yogaratnam, chief medical officer of Drug Farm, said in the release. “Obtaining rare pediatric disease designation recognizes the serious and debilitating complications of this disease and upholds our goal to provide DF-003 as the first targeted drug for potential treatment in patients afflicted with ROSAH syndrome.”