GENENTA SCIENCE S.P.A. Genenta Science announced that the U.S. Food and Drug Administration has granted Orphan Drug Designation (ODD) to Temferon(TM) for the treatment of glioblastoma multiforme (GBM). Temferon is a proprietary cell therapy designed to reprogram the tumor microenvironment by delivering immunomodulatory molecules directly to tumors. Genenta is testing Temferon in an ongoing Phase 1/2a clinical trial in newly diagnosed patients with GBM who have an unmethylated MGMT gene promoter (uMGMT-GBM).
GBM is the most common malignant primary brain tumor and the most aggressive diffuse glioma, with unmethylated MGMT gene promoters status identified in approximately 60% of the GBM population. The ODD program supports the development of treatments that address diseases affecting fewer than 200,000 people in the United States (which equates to approximately 6 cases per 10,000 population). Incentives that come with the designation include eligibility for federal grants, tax credits for qualified clinical trials, prescription drug user fee exemptions, and a seven-year marketing exclusivity period upon FDA approval.
