Ribo Receives EMA Orphan Drug Designation for siRNA Therapeutic RBD1016 Targeting Hepatitis D Virus
Key Insights
The European Medicines Agency (search) has granted Orphan Drug Designation to RBD1016, an siRNA therapeutic developed by Ribo for treating Hepatitis D Virus (search) infection.
RBD1016 utilizes Ribo's proprietary GalNAc-platform RiboGalSTAR™ to selectively silence key viral factors involved in HDV (search) infection and is currently in Phase II clinical development.
HDV (search) affects an estimated 12-20 million people globally and represents the most severe form of viral hepatitis, with limited therapeutic options currently available.
Suzhou Ribo Life Science Co., Ltd. and Ribocure Pharmaceuticals AB (search) announced that the European Medicines Agency (search) (EMA) has granted Orphan Drug Designation (ODD) to RBD1016 for the treatment of Hepatitis D Virus (search) (HDV (search)) infection. The designation represents a significant regulatory milestone for the siRNA therapeutic currently undergoing Phase II clinical development.
Regulatory Milestone for Rare Disease Treatment
The EMA's ODD is assigned to investigational therapies targeting life-threatening or chronically debilitating rare diseases that affect no more than five in 10,000 individuals in the European Union. This status provides significant regulatory and commercial incentives, enabling more rapid progression to patients in need of treatment options.
RBD1016 is built on Ribo's siRNA platform featuring the proprietary GalNAc-platform RiboGalSTAR™, which has been validated through multiple clinical studies. The siRNA drug candidate is specifically designed to selectively silence key viral factors involved in HDV (search) infection.
"This designation is a significant regulatory milestone that enhances the development and commercial potential of RBD1016," said Dr. Li-Ming Gan, Co-CEO and Global Head of R&D at Ribo. "It validates our strategy of targeting severe diseases with high unmet need through innovative RNA interference technology."
Addressing Critical Unmet Medical Need
HDV (search) represents the most severe form of viral hepatitis and occurs exclusively in individuals co-infected with hepatitis B virus (search) (HBV (search)). The virus accelerates liver disease progression, significantly increasing the risk of cirrhosis (search), liver failure (search), and hepatocellular carcinoma (search).
An estimated 12-20 million people globally are affected by HDV (search), yet limited therapeutic options are currently available. Despite its severity, HDV remains underdiagnosed and underserved by existing treatment approaches.
Clinical Development Progress
RBD1016's efficacy is currently being assessed in global Phase II clinical development. The advancement of this siRNA therapeutic represents a promising approach for patients and clinicians seeking more effective and durable treatment options for HDV (search) infection.
"We are advancing this promising therapy through clinical development, with the goal of delivering a new treatment to patients affected by this rare disease," Dr. Gan noted. "Most importantly, it represents hope for HDV (search) patients who lack effective treatment options."
The development of targeted therapies like RBD1016 offers a promising path forward for addressing the significant unmet medical need in HDV (search) treatment, potentially providing new hope for patients suffering from this severe form of viral hepatitis.