Europe Recommends Orphan Drug Designation for Alzinomics' RZ-001
Key Insights
Alzinomics (search) said the EMA's Committee for Orphan Medicinal Products (search) recommended Orphan Drug Designation for its RNA editing-based gene therapy RZ-001 in glioma (search).
COMP cited significant benefit over existing European treatments, weighing increased response rates and continuous tumor regression seen on imaging in recurrent glioblastoma (search) patients.
RZ-001 is in phase 1/2a trials in Korea and the US, with 10 patients dosed and 3 more treated under FDA Expanded Access.
Alzinomics (search) reported on September 28 that the European Medicines Agency (search)'s Committee for Orphan Medicinal Products (search) (COMP) has recommended Orphan Drug Designation for RZ-001 as a treatment for glioma (search). COMP concluded that the therapy has the potential to offer significant benefit to patients even where existing treatments are available in Europe, and forwarded the recommendation to the European Commission. European Orphan Drug Designation requires both meeting rare disease criteria and demonstrating significant benefit over existing therapies.
The committee's review focused on clinical data from patients with recurrent glioblastoma (search). According to the EMA review report, RZ-001 is in phase 1/2a trials for recurrent glioblastoma in Korea and the United States, with 10 patients treated in those trials and 3 additional patients treated under the FDA's Expanded Access program. COMP weighed increased response rates relative to approved therapies and continuous tumor regression observed in multiple patients on imaging, concluding the therapy may hold clinically meaningful advantages.
RZ-001 is a gene therapy built on Alzinomics (search)' RNA substitution enzyme technology. It cleaves telomerase reverse transcriptase (hTERT) messenger RNA expressed in cancer cells and replaces it with RNA carrying the HSV-TK suicide gene from herpes simplex virus; subsequently administered valganciclovir is converted by HSV-TK into a cytotoxic compound that inhibits DNA synthesis and induces apoptosis. Alzinomics said it plans to use the recommendation to discuss its European clinical and regulatory strategy with EMA, and noted that designation can bring protocol assistance, fee reductions and, if market authorization is later granted and conditions met, up to 10 years of market exclusivity. COMP advised the company to use protocol assistance before any marketing authorization application to define the data needed to show significant benefit. The company added that RZ-001 received FDA Advanced Regenerative Medicine Therapy designation for hepatocellular carcinoma (search) in May.
Source: The Asia Business Daily