On November 25, 2024 Rznomics, Inc. reported to secure its expanded access program (EAP) from the United States Food and Drug Administration (FDA) for RZ-001, RNA editing gene therapy product for the treatment of patients aged 18 and older with Glioblastoma (GBM) (Press release, Rznomics, NOV 25, 2024, View Source [SID1234648631]).
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Generally, the EAP also known as ‘compassionate use,’ is a pathway provided by the U.S. FDA that allows patients with serious or immediately life-threatening conditions to gain access to investigational medical products (drugs, biologics, or medical devices) outside of clinical trials.
GBM is known as the most malignant tumor in Central Nervous system with high mortality rate but lacks effective therapies. TERT promoter mutations, which are associated with TERT upregulation, are found in up to 80% of glioblastoma (GBM) patients. This increased TERT expression is strongly linked to a poor prognosis, reflecting the aggressive nature of the disease. RZ-001, the RNA replacement enzyme-based cancer gene therapy, targets and cleaves hTERT mRNA and replaces the mRNA with the therapeutic gene RNA. This induces anti-cancer activity and cytotoxic effect by reducing hTERT expression and simultaneously trans-ligating an HSVtk-encoding sequence into the reprogrammed hTERT mRNA.
"We are excited to offer RZ-001 through this EAP, providing a potential new treatment option for GBM patients with limited alternatives," said Dr. Chiocca, Professor at Harvard Medical School executive Director of the Center for Tumors of the Nervous System at Mass General Brigham Cancer Institute.
Seong-Wook Lee, PhD, CEO of Rznomics, stated, "We hope RZ-001 can serve as a good alternative for patients who have had difficulty with existing treatments." He also assured that the Rznomics team is committed to expediting the clinical development process to secure timely market authorization.
In previous releases, Rznomics noted that RZ-001 has received Fast Track designations and Phase I/IIa IND approval for RZ-001 from the FDA and the South Korean Ministry of Food and Drug Safety (MFDS) in Glioblastoma and the clinical trial has been investigating the safety, tolerability, and efficacy of RZ-001 in patients with GBM. A clinical trial has recently commenced, marking the start of RZ-001 administration.