The US Food and Drug Administration (FDA) has granted fast track designation to Rznomics’ RZ-001 for the treatment of hepatocellular carcinoma (HCC), a form of liver cancer.
This is the second such designation for the gene-therapy-based drug, following a previous status for glioblastoma in November 2023.
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The drug is undergoing Phase Ib/IIa trials for HCC and Phase I/IIa studies for glioblastoma.
Rznomics CEO Dr Seong-Wook Lee stated: “We believe this designation recognises the potential of RZ-001 as an innovative cancer therapy. We are committed to expediting its clinical development to provide effective treatment options for patients suffering from hard-to-treat cancers.”
In development using the company’s RNA-editing platform technology, Trans-splicing Ribozyme, the drug has already secured investigational new drug (IND) approval from South Korea’s Ministry of Food and Drug Safety and the US regulator.
RZ-001 benefits from the agency’s fast track programme, which is designed to expedite the development and review of drugs targeting serious or life-threatening conditions.
The designation allows for accelerated approval processes, more frequent communication with the agency and the possibility of a rolling review during the biologics licence application phase.
In addition to its fast track status for HCC, RZ-001 has been approved for compassionate use under the US regulator’s Expanded Access Program (EAP) for patients with glioblastoma.
This programme is being carried out at Harvard University Hospital, providing access to investigational treatment outside clinical trials.
Rznomics, which originated in the laboratory of Professor Seong-Wook Lee in Dankook University’s Department of Bioconvergence Engineering, is focused on developing new RNA-based gene therapeutic bio-drugs for cancer and other incurable conditions.
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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