RARE Daily

FDA Grants Rare Pediatric Disease Designation to Drug Farm for ROSAH Syndrome Treatment

January 17, 2024

Rare Daily Staff

The U.S. Food and Drug Administration has granted Rare Pediatric Disease designation for Drug Farm’s alpha-kinase 1 inhibitor, DF-003, to treat patients with ROSAH syndrome.

ROSAH (retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis and headache) syndrome is a rare, autosomal dominant autoinflammatory disease named according to the characteristic symptoms exhibited by affected patients. The disease is caused by a genetic gain-of-function mutation in the alpha-kinase 1 gene (ALPK1). The most common presenting symptom is a progressive decline in visual acuity that typically begins before 20 years of age, with ophthalmologic examination often revealing optic disc elevation, uveitis, and retinal nerve degeneration. Most ROSAH patients also exhibit inflammatory features such as non-infectious low-grade fevers, arthralgia, headaches, and persistently elevated levels of inflammatory cytokines including tumor necrosis factor α (TNFα), interleukin 6 (IL-6), and IL-1β.

DF-003 is currently being evaluated in a phase 1 trial 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.”

Rare Pediatric Disease designation is granted by the FDA for serious or life-threatening diseases which affect fewer than 200,000 people in the United States and in which the serious or life-threatening manifestations primarily affect individuals less than 18 years of age. If a New Drug Application for DF-003 to treat ROSAH Syndrome is approved by the FDA, Drug Farm may be eligible to receive a Priority Review Voucher that can be redeemed to receive a priority review for any subsequent marketing application or may be sold or transferred. The FDA has implemented this program to encourage development of new drugs for treatment of rare pediatric diseases.

Photo: Jeysen Yogaratnam, chief medical officer, Drug Farm

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