Drug Farm Receives Rare Pediatric Disease Designation from the U.S. FDA for DF-003 to Treat ROSAH Syndrome
Drug Farm , a clinical-stage biopharmaceutical company announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) Designation for the company’s alpha-kinase 1 (ALPK1) inhibitor, DF-003, to treat patients with ROSAH (retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis and headache) Syndrome.
- Drug Farm , a clinical-stage biopharmaceutical company announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) Designation for the company’s alpha-kinase 1 (ALPK1) inhibitor, DF-003, to treat patients with ROSAH (retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis and headache) Syndrome.
- DF-003 is currently being evaluated in a Phase 1 trial ( NCT05997641 ) to assess safety and pharmacokinetics in normal healthy volunteers.
- “Pediatric patients living with ROSAH Syndrome face a significant unmet need with limited options to treat vision loss,” said Jeysen Yogaratnam, Chief Medical Officer, Drug Farm.
- “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.”