Alzinomics RZ-001 Recommended for Orphan Drug Designation in Europe

EMA Committee for Orphan Medicinal Products
Recognizes "Significant Benefit" Based on Clinical Data in Recurrent Glioblastoma
Assesses Potential Clinical Advantages Over Existing Therapies
"Expecting Support for European Clinical and Regulatory Strategies"

Alzinomics announced on September 28 that its RNA editing and correction technology-based anticancer gene therapy, RZ-001, has received a recommendation for Orphan Drug Designation as a treatment for glioma from the Committee for Orphan Medicinal Products (COMP) under the European Medicines Agency (EMA).

COMP determined that RZ-001 has the potential to offer “significant benefit” to patients even in the presence of existing treatments in Europe, and therefore recommended Orphan Drug Designation to the European Commission. Orphan Drug Designation in Europe not only requires meeting the criteria for rare diseases, but also mandates demonstrating significant benefits over existing therapies.

Algonomics company logo image. Algonomics

In its recent review, COMP primarily evaluated the clinical data of RZ-001 observed in patients with recurrent glioblastoma. According to EMA’s review report, RZ-001 is currently undergoing phase 1/2a clinical trials for recurrent glioblastoma patients in both Korea and the United States. In these clinical trials, the therapy was administered to 10 individuals, and an additional 3 received it under the U.S. Food and Drug Administration (FDA)’s Expanded Access (compassionate use) program.

COMP acknowledged the therapeutic potential of RZ-001 based on the clinical responses observed in patients with recurrent glioblastoma. It reviewed the increased response rates in patients compared to approved therapies and concluded that the therapy may have clinically meaningful advantages. The committee also put significant weight on the continuous tumor regression observed in multiple patients during imaging evaluations.

RZ-001 is a gene therapy product that leverages Alzinomics’ RNA substitution enzyme technology. It targets and cleaves the telomerase reverse transcriptase (hTERT) messenger RNA, which is expressed in cancer cells, and replaces it with RNA containing the HSV-TK suicide gene derived from herpes simplex virus. Subsequently, the administered valganciclovir is converted into a cytotoxic compound by HSV-TK, thereby inhibiting DNA synthesis in cancer cells and inducing apoptosis.

Alzinomics plans to use this Orphan Drug Designation recommendation as a springboard for active and specific discussions with EMA regarding its clinical and regulatory strategy in Europe. Upon Orphan Drug Designation, the company may benefit from various regulatory advantages, such as protocol assistance from EMA for clinical and regulatory planning, as well as fee reductions. If the product attains market authorization in the future and fulfills related conditions, it could also gain up to 10 years of market exclusivity.

COMP recommended that, prior to a market authorization application, Alzinomics utilize protocol assistance and consult with EMA in advance to determine the data necessary to demonstrate that RZ-001, as an orphan drug, offers significant benefit.

Bomi Kim, Head of the New Drug Development Center at Alzinomics, stated, “The significance lies in the fact that the early clinical results observed in actual recurrent glioblastoma patients were recognized by European regulatory authorities as holding the potential for clinically meaningful advantages over existing therapies. We plan to pursue global clinical development and regulatory strategies even more actively going forward.”

Seonguk Lee, CEO of Alzinomics, commented, “Following our designation as an Advanced Regenerative Medicine Therapy for hepatocellular carcinoma by the FDA this May, regulatory authorities have now also recognized the clinical potential of RZ-001. We will continue to pursue expansion of indications as well as business development and value enhancement for RZ-001.”

This content was produced with the assistance of AI translation services.