Sunday, February 9, 2025

Rznomics’ HCC Treatment Receives Orphan Drug Designation by FDA

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Rznomics’ drug, RZ-001, has been designated as an orphan drug by the FDA, a status that could lead to seven years of marketing exclusivity alongside approval. Additional benefits of this designation include exemption from user fees and eligibility for tax credits. The drug will also receive regulatory assistance from the Office of Orphan Products Development (OOPD).

RZ-001 is constructed to suppress hTERT expression, which is often overexpressed in cancer cells. This effect is combined with a cytotoxic effect achieved through the trans-ligating of an HSVtk-encoding sequence. Preclinical animal models have demonstrated that this methodology effectively provokes immune cell infiltration into hepatocellular carcinoma (HCC) tumors.

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FDA and MFDS Approvals as Orphan Drug Designation and IND Clearance

The FDA orphan drug designation emphasizes the potential of Rznomics’ pipeline to promptly address the current unmet medical needs of HCC patients, according to Rznomics CEO and founder Seong-Wook Lee. The company has also received Investigational New Drug (IND) application clearance from the US FDA and the South Korean Ministry of Food and Drug Safety (MFDS) for Phase I/IIa studies of RZ-001 in HCC.

The upcoming trial will be a dose escalation/expansion study to evaluate the safety, tolerability, and efficacy of RZ-001 in HCC patients without extrahepatic metastasis. RZ-001 has also obtained Phase I/IIa IND approvals for use in glioblastoma multiforme (GBM) in the US and South Korea, in addition to its fast-track designation.

Orphan Drug

Partnership with Roche and Australian Trial for Genetic Retinal Disease Treatment

Rznomics has recently entered into a clinical partnership with Roche to analyze RZ-001 along with Roche’s atezolizumab. In January, Rznomics received approval for the Phase I clinical trial review of RZ-004, another asset, in Australia. RZ-004 is being developed to treat genetic retinal diseases, including Retinitis Pigmentosa. The review is currently being conducted by the Office of the Gene Technology Regulator (OGTR) in Australia.

The orphan drug designation of RZ-001 highlights its potential for addressing the needs of HCC patients. The upcoming trials, collaborations, and additional drug developments further strengthen Rznomics’ commitment to advancing medical solutions for various diseases.


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