[Edaily Reporter KIM SAE-MI ] #Rznomics Inc. announced on the 22nd that its anti-cancer gene therapy candidate “RZ-001” has received Orphan Drug Designation from the European Commission (EC) for the treatment of glioma.

This designation follows the positive opinion adopted last September by the Committee for Orphan Medicinal Products (COMP) under the European Medicines Agency (EMA). With the EC’s final decision, the process for RZ-001’s designation as an orphan drug in Europe has been completed.

The European Orphan Drug Designation is a program that supports the development of treatments for diseases with a small patient population and limited treatment options. Designated drugs are eligible for benefits such as regulatory advice and fee reductions during the development process. In principle, once marketing authorization is granted and relevant requirements are met, the drug is also granted a 10-year market exclusivity period.

Previously, RZ-001 received Orphan Drug designation and Fast Track designation from the U.S. Food and Drug Administration (FDA) for hepatocellular carcinoma and glioblastoma, respectively. In May, it also secured Regenerative Medicine Advanced Therapy (RMAT) designation for the hepatocellular carcinoma indication.

Rznomics Inc. plans to advance the global clinical development of RZ-001 based on the regulatory support secured in the U.S. and Europe. In addition to hepatocellular carcinoma and glioblastoma, which are currently under development, the company is seeking to expand indications to other solid tumors and explore commercialization opportunities such as technology transfer and joint development.

Lee Seong-wook, CEO of Rznomics Inc., stated, “Glioblastoma has a poor prognosis and represents a significant unmet medical need,” adding, “Following the European Commission’s designation as an orphan drug, we will do our utmost to ensure that RZ-001 becomes a new treatment option for patients with intractable cancers.”