#Sweden #Gothenburg #Ribo Life Science #RBD1016 #EMA Orphan Drug

Ribo's RBD1016 Receives Orphan Drug Designation

In a recent announcement, Suzhou Ribo Life Science Co. and Ribocure Pharmaceuticals AB (Ribo) revealed that the European Medicines Agency (EMA) has granted orphan drug designation to their therapeutic candidate, RBD1016, which targets the infection caused by the Delta hepatitis virus (HDV). This significant achievement highlights the increasing recognition of the need for safer and more effective therapies for patients afflicted by rare conditions.

Understanding Orphan Drug Designation

The orphan drug designation from the EMA is a crucial designation aimed at encouraging the development of treatments for rare diseases that affect fewer than five in 10,000 individuals in the European Union. These designations come with various regulatory and commercial incentives designed to expedite the development process of potentially life-saving treatments. By providing RBD1016 with this status, Ribo is poised to advance the clinical investigation more rapidly, ultimately leading to earlier availability for patients in need.

RBD1016 and Its Mechanism of Action

RBD1016 uses a pARNi platform integrated with Ribo’s proprietary GalNAc RiboGalSTARTM technology, which has undergone validation through various clinical studies. Specifically, RBD1016 aims to selectively silence key viral factors associated with HDV infection. Currently, the effectiveness of this innovative drug candidate is being evaluated in a Phase II global clinical development program, emphasizing its potential to significantly alter the treatment landscape for HDV.

Dr. Li-Ming Gan, Co-CEO and Global Head of R&D at Ribo, stated, “This designation represents an important regulatory milestone that enhances the development and commercialization potential of RBD1016. It validates our strategy of targeting serious diseases with 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 suffering from this rare disease. Importantly, it represents hope for patients with HDV who currently have no effective therapeutic options available.”

The Urgency of Addressing HDV Infection

Delta hepatitis (HDV) is considered the most severe form of viral hepatitis and occurs only in individuals co-infected with the hepatitis B virus (HBV). The presence of HDV accelerates liver disease progression, significantly increasing the risk of complications such as cirrhosis, liver failure, and hepatocellular carcinoma. It is estimated that between 12 to 20 million individuals worldwide are affected by HDV, highlighting the urgent need for effective treatment options. Unfortunately, despite its severity, HDV remains underdiagnosed and underserved, with limited therapeutic options currently available.

Ribo's RBD1016 presents a promising avenue for patients and healthcare practitioners searching for more effective and long-lasting therapeutic alternatives in the fight against HDV.

About Ribo

Founded in China, Suzhou Ribo Life Science Co. Ltd. is at the forefront of advancing RNA interference technology and industrializing oligonucleotide-based medicines. The company has developed a comprehensive therapeutic platform incorporating all necessary technologies for successful drug development and has established a robust pipeline of pARNi products. Ribocure Pharmaceuticals AB, located in Gothenburg, Sweden, serves as Ribo's global R&D hub.

For further details about Ribo’s innovative developments and therapeutic pipelines, visit their websites at www.ribolia.com and www.ribocure.com.