Suzhou Ribo Life Science Co., Ltd. and Ribocure Pharmaceuticals AB announced today that the European Medicines Agency (EMA) has granted orphan drug designation to RBD1016, a therapeutic small interfering RNA (siRNA) intended for treating Hepatitis D Virus (HDV) infection. This designation is reserved for investigational therapies aimed at rare and potentially life-threatening diseases that affect fewer than five in 10,000 individuals within the European Union.
The orphan drug designation provides significant regulatory and commercial incentives, facilitating expedited development for patients suffering from these conditions. The Ribo siRNA platform, featuring its proprietary GalNAc delivery system known as RiboGalSTARTTM, has achieved validation through multiple clinical studies.
RBD1016 is among the company”s programs where the siRNA candidate is engineered to selectively silence key viral factors associated with HDV infection. The efficacy of RBD1016 is currently under investigation in a Phase II global clinical development program.
“This designation represents a significant regulatory milestone that enhances the development and commercial potential of RBD1016,” stated Dr. Li-Ming Gan, co-CEO and Global Head of R&D at Ribo. “It validates our strategy to tackle severe diseases with high unmet needs through innovative RNA interference technology. We are advancing this promising therapy through clinical development, aiming to provide a new treatment option for patients affected by this rare disease. Most importantly, it represents hope for HDV patients lacking effective treatment alternatives.”
Hepatitis D Virus is the most severe form of viral hepatitis and occurs exclusively in individuals co-infected with Hepatitis B Virus (HBV). HDV accelerates the progression of liver disease, substantially increasing the risk of cirrhosis, liver failure, and hepatocellular carcinoma. It is estimated that between 12 million and 20 million people globally are affected by HDV, with currently available therapeutic options being limited. Despite its severity, HDV remains underdiagnosed and undertreated. The development of targeted therapies like RBD1016 offers a promising future for patients and healthcare professionals seeking more effective and lasting treatment options.
Suzhou Ribo Life Science Co., Ltd. is at the forefront of advancing RNA interference technology and the industrialization of oligonucleotide drugs in China. The company has established a therapeutic oligonucleotide platform that vertically integrates a comprehensive set of technologies and built a global integrated drug development capability alongside a robust siRNA production line. Ribocure Pharmaceuticals AB serves as Ribo”s international R&D center, headquartered in Gothenburg, Sweden. For further information, visit www.ribolia.com and www.ribocure.com.
