This designation could lead to seven years of marketing exclusivity on receipt of an approval, as well as exemption from user fees and eligibility for tax credits.
Furthermore, the drug will receive regulatory assistance from Office of Orphan Products Development (OOPD) of the regulator.
RZ-001 is designed to act on and suppress hTERT expression, which is overexpressed in cancer cells, while also eliciting a cytotoxic effect through the trans ligating of an HSVtk-encoding sequence.
Preclinical animal models have shown that this method effectively induces immune cell infiltration into HCC tumours.
Rznomics CEO and founder Seong-Wook Lee said: “This FDA orphan drug designation further underlines the potential of our pipeline to expeditiously address the current unmet medical needs of patients with hepatocellular carcinoma.”
The company has received Investigational New Drug (IND) application clearance for Phase I/IIa studies of RZ-001 in HCC from the US FDA and the South Korean Ministry of Food and Drug Safety (MFDS).
The prospective trial will be a dose escalation/expansion study to assess the safety, tolerability, and efficacy of RZ-001 in HCC patients without extrahepatic metastasis.
In addition to HCC, Rznomics obtained Phase I/IIa IND approvals for RZ-001 in glioblastoma multiforme (GBM) in both South Korea and the US, as well as fast track designation.
Recently, the company entered into a clinical partnership with Roche to analyse RZ-001 along with latter’s atezolizumab.
Rznomics received approval in January for the Phase I clinical trial review of another asset, RZ-004, in Australia.
RZ-004 is intended to treat for genetic retinal diseases, including Retinitis Pigmentosa.
The review is being conducted by the Office of the Gene Technology Regulator (OGTR) in Australia.