EC final decision follows EMA's positive opinion in September, targeting glioma
Benefits include clinical trial design consultation, fee reductions, and a 10-year market exclusivity upon meeting post-approval requirements
Alzinova's (476830) oncology gene therapy candidate 'RZ-001' has been designated as an orphan medicinal product in Europe. As early-stage clinical trials are underway for recurrent glioblastoma, the company has secured regulatory support necessary for development and approval processes in Europe as well.
Alzinova announced on the 22nd that RZ-001 received orphan drug designation from the European Commission (EC) for the treatment of glioma. This final decision follows a positive opinion issued in September by the Committee for Orphan Medicinal Products (COMP) under the European Medicines Agency (EMA). Glioblastoma is a type of glioma.
The European orphan drug designation is a system designed to encourage the development of treatments for rare and serious diseases. Designated candidates are eligible for scientific advice on clinical trial design and fee waivers depending on the applicant and service type. Upon receiving marketing authorization and meeting the maintenance requirements for orphan drug status, the therapy can in principle be granted 10 years of market exclusivity. However, the designation itself does not permit sales or immediately initiate exclusivity.
RZ-001 targets the messenger RNA (mRNA) of human telomerase reverse transcriptase (hTERT), which is expressed in cancer cells. Using an RNA-cleaving enzyme, it is designed to replace a portion of the target RNA with therapeutic RNA and induce the production of an enzyme derived from the herpes simplex virus (HSV-TK) inside cancer cells. Subsequently, co-administered valganciclovir is converted into ganciclovir, which is activated by HSV-TK and other factors to induce cancer cell death.
Interim data from a phase 1/2a clinical trial in patients with hTERT-positive recurrent glioblastoma were used in the designation review. According to the company, the COMP evaluated that RZ-001 has the potential to provide clinically meaningful benefits to patients even where existing therapies are available.
In June, interim results from 10 patients enrolled and dosed at the time were unveiled at the Asian Society for Neuro-Oncology (ASNO) meeting. According to company IR materials, grade 3 or higher adverse events, regardless of causality with the drug, occurred in 5 patients (50%). Grade 3 adverse events assessed as related to treatment were observed in 1 patient (10%). There were no grade 4 or higher adverse events, and the company stated that no dose-limiting toxicities were reported. Disease control exceeding six months was observed in some patients.
These results stem from an interim analysis of a small patient cohort. Professor Kim Jae-yong of the Department of Neurosurgery at Seoul National University Bundang Hospital, who presented the findings, stated he expects the clinical value to be more clearly verified through additional patient enrollment and long-term follow-up.
In the United States, RZ-001 has secured orphan drug and fast track designations for both hepatocellular carcinoma and glioblastoma indications. In May of this year, it was also designated as a Regenerative Medicine Advanced Therapy (RMAT) for the hepatocellular carcinoma indication.
Building on the regulatory benefits secured in the U.S. and Europe, Alzinova plans to continue its clinical development while exploring opportunities for label expansion, technology licensing, and co-development.
