FDA grants rare pediatric disease designation to alpha kinase inhibitor for ROSAH syndrome

The FDA has granted rare pediatric disease designation to an alpha-kinase 1 inhibitor to treat patients with retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis and headache, or ROSAH, syndrome.

According to a press release from developer Drug Farm, DF-003 is currently being evaluated in a phase 1 clinical trial assessing safety and pharmacokinetics in healthy volunteers.

The rare pediatric disease designation is granted by the FDA for a serious or life-threatening condition that affects fewer than 200,000 people in the United States and primarily affects those aged younger than 18 years.

“Pediatric patients living with ROSAH syndrome face a significant unmet need, with limited options to treat vision loss,” Jeysen Yogaratnam, chief medical officer, Drug Farm, stated in the release. “Obtaining rare pediatric disease designation recognizes the serious and debilitating complications of this rare disease and upholds our goal to provide DF-003 as the first targeted drug for potential treatment in patients afflicted with ROSAH syndrome.”