Sino-US biotech company Drug Farm has announced that it has received Rare Pediatric Disease Designation (RPDD) from the US Food and Drug Administration (FDA) for its first-in-class drug candidate DF-003, which is being investigated as a potential treatment for the ROSAH (retinal malnutrition, optic edema, splenomegaly, anhidrosis, and headache) syndrome.
DF-003 works by inhibiting the activities of ALPK1 and ALPK1 mutations that are responsible for causing ROSAH syndrome. The drug is currently in a Phase I study, assessing its safety and pharmacokinetics in healthy subjects.
ROSAH syndrome is a rare autosomal dominant inherited autoimmune inflammatory disease characterized by its distinct set of symptoms. The condition arises from a highly active genetic mutation in ALPK1, with the most prevalent symptom being a progressive decline in vision, often beginning before the age of 20. Patients typically exhibit elevated optic discs, uveitis, and retinal nerve degeneration upon ophthalmic examination. In addition to vision problems, ROSAH patients often present with inflammatory symptoms such as low-grade fever, joint pain, headache, and increased levels of inflammatory cytokines like tumor necrosis factor α (TNF-α), interleukin-6, and IL-1β.
The RPDD, applicable to drugs that treat serious or life-threatening diseases affecting children under 18 years old and fewer than 200,000 patients in the US, could lead to a Priority Review Voucher (PRV) for Drug Farm if the company obtains marketing approval for DF-003 for this indication. The voucher can potentially be transferred for a value of USD 100 million.- Flcube.com
