Temferon™ Granted Orphan Drug Designation by European Commission for Glioma Treatment
Friday, June 30, 2023
Genenta Science (NASDAQ: GNTA) has recently received Orphan Drug Designation (ODD) from the European Commission for their product Temferon™, which is being developed for the treatment of glioma, specifically Glioblastoma Multiforme (GBM). ODD is granted to drugs that target rare conditions affecting a small number of individuals in the European Union (EU) and provide significant benefits in the absence of satisfactory treatment options. This designation comes with various incentives, including ten years of market exclusivity after obtaining marketing authorization.
Pierluigi Paracchi, the CEO of Genenta, expressed his satisfaction with the European Medicines Agency's Committee's review of the ODD application for Temferon. He emphasized the potential significant benefit of Temferon in treating GBM and its role in advancing Genenta's cell therapy-based technology platform for solid tumors. Paracchi also mentioned that Temferon had previously received orphan drug designation from the US Food and Drug Administration in March 2023 for GBM treatment. Encouraging results from ongoing phase 1/2a trials of Temferon in newly diagnosed patients with unmethylated MGMT gene promoter have shown overall survival at two years exceeding the median overall survival reported in published studies.
GBM is the most common and aggressive malignant primary brain tumor, with approximately 60% of GBM cases exhibiting unmethylated MGMT promoter status.
Temferon, Genenta's leading product, involves modifying the patient's own stem progenitor cells using their platform to express interferon alpha (IFNa) within solid tumors. IFNa is a well-known immunomodulatory protein with a history of use in cancer treatment, albeit with limited application due to systemic toxicity. Genenta's platform aims to selectively deliver therapeutic activity within the solid tumor, bypassing systemic toxicity concerns. Preclinical experiments have demonstrated that Temferon can disrupt tumor-induced tolerance, allowing the immune system to recognize the tumor and mount a durable immune response.
