The US Food and Drug Administration (FDA) has accepted DF-003 into the FDA Rare Disease Evidence Principles Process (RDEP) for the potential treatment of ROSAH syndrome.
ROSAH (retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis, and headache) syndrome is a rare autoinflammatory genetic disorder caused by mutations in the ALPK1 gene. The disease is characterized by retinal degeneration, optic nerve edema, systemic inflammation, and progressive vision loss. There are currently no approved therapies for ROSAH syndrome and treatment is limited to symptom management (i.e., anti-inflammatory agents, poor vision assistance).
DF-003 is a proprietary, first-in-class therapy designed to inhibit ALPK1 and variants of ALPK1 activity. A phase 1 clinical trial (NCT05997641) has been completed in normal healthy volunteers and is now recruiting patients with ROSAH syndrome in a phase 1b study (NCT06395285).
The RDEP program is designed to facilitate early communication between the agency and sponsors developing therapies for rare diseases. The program allows the FDA to discuss regulatory strategies, clinical evidence development, and innovative approaches to generating the evidence needed to support regulatory submissions with selected sponsors.
For more information, visit the developers of DF-003 (Drug Farm) or a recent review in Nature Communications on how DF-003 was developed.
To learn more about rare eye disorders, visit https://checkrare.com/diseases/ophthalmology-eye-diseases/
