ALBANY, N.Y. & SHANGHAI–(BUSINESS WIRE)–Drug Farm today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to DF-003, the company’s investigational small molecule inhibitor targeting ALPK1, for the treatment of patients with ROSAH syndrome, a rare, genetically driven systemic autoinflammatory disease with significant unmet medical need.
ROSAH syndrome (Retinal dystrophy, Optic nerve edema, Splenomegaly, Anhidrosis, and Headache) is a progressive multisystem disorder driven by disease-causing mutations in ALPK1, leading to chronic inflammation and vision-threatening complications. Currently, there are no approved disease-modifying therapies for ROSAH syndrome.
“Receiving Orphan Drug Designation for DF-003 represents an important regulatory milestone and underscores the urgent need for targeted therapies in ROSAH syndrome,” said Henri Lichenstein, Ph.D., Chief Executive Officer at Drug Farm. “We believe DF-003 has the potential to address the underlying inflammatory drivers of this disease and deliver meaningful clinical benefit to patients.”
Orphan Drug Designation provides several key incentives to support the development of therapies for rare diseases, including:
- Up to seven years of market exclusivity in the United States upon approval
- Eligibility for FDA fee reductions or waivers
- Access to potential tax credits for qualified clinical trial costs
- Enhanced regulatory guidance throughout the development process
DF-003 is a first-in-class ALPK1 inhibitor designed to correct the genetic root cause of ROSAH syndrome by inhibiting disease-causing ALPK1 mutations. Preclinical data and early clinical observations support its potential to reduce systemic inflammation associated with ROSAH syndrome.
Drug Farm is currently advancing DF-003 in clinical development, with ongoing studies evaluating safety, pharmacokinetics, pharmacodynamic biomarkers and efficacy parameters relevant to disease activity.
About DF-003
DF-003 is a proprietary, first-in-class drug developed by Drug Farm that inhibits the activity of ALPK1 and variants of ALPK1 which cause ROSAH syndrome. DF-003 has therapeutic potential for ROSAH syndrome, as well as heart and kidney diseases, as the drug has shown efficacy in preclinical models of these indications. DF-003 has completed a Phase 1 clinical trial (NCT05997641) in normal healthy volunteers and is now accruing patients with ROSAH syndrome in a Phase 1b trial (NCT06395285).
About ROSAH Syndrome
ROSAH (retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis, and headache) syndrome is a rare, autosomal dominant autoinflammatory genetic disease caused by activating mutations in the ALPK1 gene. The disease is characterized by progressive visual loss, optic nerve and retinal pathology, and systemic inflammatory manifestations, including elevated pro-inflammatory cytokines. Symptoms often begin in childhood or early adulthood, and there are currently no approved disease-modifying therapies for ROSAH syndrome.
About Drug Farm
Drug Farm is a private biotechnology company developing innovative treatments targeting innate immunity for hepatitis B, heart and kidney diseases, and ROSAH syndrome. Drug Farm’s unique IDInVivo platform combines breakthrough technologies in genetics and artificial intelligence to identify and validate novel drug targets directly in living animals with intact immune systems. Using this platform, Drug Farm is advancing multiple first-in-class drug candidates into clinical development.
For more information, please visit: https://www.drug-farm.com
Contacts
United States:
Henri Lichenstein, Ph.D.
Chief Executive Officer
Email: [email protected]
China:
Tony Xu, Ph.D.
Co-founder and Chief Operating Officer
Email: [email protected]
