SEONGNAM, South Korea, Oct. 22, 2026 /PRNewswire/ -- Rznomics Inc., a biopharmaceutical company specializing in the development of RNA-based gene therapies, announced today that the European Commission (EC) has granted Orphan Medicinal Product Designation to 'taspitimagene advec(RZ-001)', its RNA-editing gene therapy for the treatment of glioma.
The designation follows the positive opinion adopted in September 2026 by the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency's (EMA). This marks RZ-001's first regulatory designation in Europe and further strengthens its development foundation across the two major markets of the United States and Europe.
Orphan Medicinal Product Designation is intended to encourage the development of medicines for rare, serious, or life-threatening conditions. The program offers regulatory and development benefits, including the possibility of market exclusivity following approval, reduced development fees, and support to enhance clinical development efficiency for orphan drugs.
"Following its designation in the United States, RZ-001's orphan designation in Europe provides further positive momentum for its development," said Bomi Kim, Head of the Drug Development Center at Rznomics. "Building on this milestone, we will continue to communicate the value of RZ-001 to potential global partners."
"Glioblastoma has a poor prognosis and remains an area of significant unmet medical need," added Dr. Seong-Wook Lee, CEO of Rznomics. "With the European Commission's orphan designation of RZ-001, we will continue our efforts to develop RZ-001 as a potential new treatment option for patients with difficult-to-treat cancers."
RZ-001 has also secured several regulatory designations from the U.S. Food and Drug Administration (FDA), supporting its development prospects and potential partnering value. The FDA has granted Orphan Drug Designation (ODD) and Fast Track designation for RZ-001 for treatment of hepatocellular carcinoma (HCC) and glioblastoma (GBM). In addition, RZ-001 received Regenerative Medicine Advanced Therapy (RMAT) designation for HCC in May 2026.
Rznomics aims to advance global partnerships and expand the commercial potential of RZ-001. In addition to HCC and GBM, which are currently under clinical development, the company is pursuing expansion into additional solid tumor indications.
About Glioblastoma (GBM)
Glioblastoma is the most common and aggressive primary malignant brain tumor in adults, classified as a WHO Grade IV tumor. Characterized by rapid progression, high recurrence rates, and resistance to standard therapies, recurrent glioblastoma remains an area of significant unmet medical need with limited effective therapeutic options available globally.
About Orphan Medicinal Product Designation in the European Union
Orphan Medicinal Product Designation in the European Union is intended to encourage the development of medicines for rare, serious, or life-threatening conditions. Designated products may benefit from regulatory incentives, including protocol assistance and fee reductions. Following marketing authorization, eligible products may receive ten years of market exclusivity, which may be extended to twelve years in certain circumstances.
The EU orphan designation assessment considers several factors, including the rarity and severity of the disease, the rationale for developing the medicinal product, and the availability of existing treatments. Where approved treatment methods already exist, evidence supporting the potential for the investigational product to provide a "significant benefit" to patients is an important consideration.
How RZ-001 (taspitimagene advec) Works
RZ-001 utilizes Rznomics' proprietary RNA trans-splicing ribozyme technology. The ribozyme embedded in RZ-001 selectively recognizes and cleaves hTERT mRNA expressed in cancer cells, replacing it with an RNA strand containing a suicide gene (HSV-TK) derived from the herpes simplex virus. Subsequently, the orally administered antiviral prodrug valganciclovir, is converted to ganciclovir and further activated by HSV-TK, generating cytotoxic metabolites that inhibit viral and cellular DNA synthesis and selectively induce apoptosis in cancer cells.
About Rznomics Inc.
Rznomics is a clinical-stage biopharmaceutical company based in South Korea focused on developing RNA-based gene therapies. The company's proprietary trans-splicing ribozyme platform enables precise RNA editing and has broad applicability across multiple indications. The company signed a research collaboration and license agreement with the global pharmaceutical company Eli Lilly in May 2025 for the development of a novel RNA editing therapeutic and listed on the KOSDAQ market in December 2025. (KOSDAQ 476830)
For more information, please visit www.rznomics.com
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