[Edaily Reporter KIM SAE-MI ] #Rznomics Inc.’s RNA-based anticancer gene therapy “RZ-001” (taspitimagene advec) has passed the Committee for Orphan Medicinal Products (COMP) review for designation as an orphan drug in Europe.

Rznomics Inc. announced on the 28th that the Committee for Orphan Medicinal Products (COMP), under the European Medicines Agency (EMA), adopted a positive opinion recommending that RZ-001 be designated as an orphan drug for the treatment of glioma.

COMP’s positive opinion is part of the Orphan Drug designation process and will now proceed to the final decision-making process by the European Commission (EC).

According to the company, COMP determined that RZ-001 meets the requirements for Orphan Drug Designation in Europe. In particular, COMP reviewed RZ-001’s early clinical data regarding the “significant benefit” requirement—a key criterion for Orphan Drug Designation—especially given that there are already approved treatments for this condition.

Rznomics Inc. reported that the COMP recognized RZ-001’s therapeutic potential based on clinical responses observed in patients with recurrent glioblastoma (GBM). The company explained that the potential for clinically meaningful benefits—based on observed responses compared to existing treatments—was acknowledged at the orphan drug designation stage.

Glioblastoma is a highly malignant brain tumor among gliomas. RZ-001 is currently undergoing a Phase 1/2a clinical trial in South Korea and the United States for patients with hTERT-positive recurrent glioblastoma. To date, the drug has been administered to 10 patients in clinical trials and to 3 patients through the U.S. Food and Drug Administration’s (FDA) Expanded Access Program (EAP).

RZ-001 is an adenovirus type 5-based gene therapy that utilizes Rznomics Inc.’s RNA trans-splicing ribozyme technology. It incorporates an RNA trans-splicing ribozyme that targets the messenger ribonucleic acid (mRNA) of human telomerase reverse transcriptase (hTERT), which is expressed in cancer cells, along with the thymidine kinase (HSV-TK) gene derived from the herpes simplex virus.

If RZ-001 is ultimately designated as a European Orphan Drug, it will be eligible for protocol assistance from the EMA during the development process and benefits such as reductions in certain regulatory fees. Upon obtaining marketing authorization and meeting the relevant requirements, the product may be granted up to 10 years of market exclusivity in the EU for the designated rare disease indication.

COMP recommended that, during future clinical development, the company utilize the EMA’s protocol assistance to engage in advance consultations regarding clinical trial design, quality, non-clinical and clinical development, and data necessary to demonstrate significant benefits over existing treatments.

Lee Seong-wook, CEO of Rznomics Inc., stated, “Following the Regenerative Medicine Advanced Therapy (RMAT) designation for hepatocellular carcinoma (HCC) granted by the FDA in May of this year, the clinical potential of RZ-001 has now been evaluated by regulatory authorities,” adding, “We will move forward with expanding indications and advancing business development.”