FDA Gives Orphan Drug Designation to Temferon for Glioblastoma Multiforme


The FDA has granted the cell therapy temferon an orphan drug designation to treat patients with brain cancer.

The FDA provided an orphan drug status for the cell therapy Temferon for the treatment of patients with glioblastoma multiforme (GBM), according to a press release from the drug manufacturer, Genenta Science.1

In the press release, the clinical-stage immuno-oncology company explained that the orphan drug designation will allow for further development of temferon for the most common malignant primary brain that shows up in approximately 200,000 patients.

About 60% of the GBM population has unmethylated MGMT promoter status, which Temferon targets. Moreover, Temferon release the known antitumor agent IFN-α by altering Tie-2-expressing monocytes, which restores the immune system and offsets tumor growth without the high amount of toxicity that IFN-α is associated with as a single-agent.

brain cancer, GBM, Glioblastoma Multiforme, Temferon

"We expect that the FDA’s decision to grant [an] orphan drug designation to Temferon will enhance the development of our cell therapy, which we believe has the potential to address the unmet medical need of patients and strengthen our clinical program," explained Pierluigi Paracchi, chief executive officer of Genenta, in a press release announcing the designation. "The orphan drug designation program highlights the significant need for an efficacious therapy for patients suffering from glioblastoma multiforme."

The safety and evaluation of Temferon is being analyzed in a phase 1/2a open-label, multicenter, therapeutic-exploratory, dose-escalation, prospective study (NCT03866109) that will administer a single injection of Temferon to 27 patients with GBM who have an unmethylated MGMT promoter.2 The primary outcome measure of the trial is the tolerability and safety of Temferon over the first 90 days after administration of the cell therapy and determine the incidence of adverse events in this time period.

Secondary outcomes the study are looking at the long-term tolerability and safety of Temferon, the proportion of patients achieving hematologic recovery by day +30, and to determine the maximum tolerated dose of Temferon.

Eligible patients for the trial had to have undergone complete or partial tumor resection, be eligible for radiotherapy, have a life expectancy of at least 6 months, and a Karnofsky performance score of at least 70. At 20 days of administration of Temferon, patients on the therapy need to have an acceptable cardiac, renal, hepatic, and pulmonary function, a left ventricular ejection fraction of at least 45%, and absence of severe pulmonary hypertension. Moreover, the researchers are looking to determine the short-term tolerability of the therapy as defined by stable blood counts, absence of cytopenias, absence of significant organ toxicities greater than grade 2, and the absence of replication competent lentivirus.

The data cutoff of the trial was on October 15, 2021, and consisted of a median follow up of 267 days (range, 60-749) with patients assigned to 7 different cohorts (n = 3 each) differing in a dosing range of 0.5 to 4.0 x 106/kg, and conditioning regimen of carmustine and thiotepa or busulfan and thiotepa. Patients in cohorts 1-3 received Temferon at a dose of 0.5-2.0 x 106/kg with an average vector copy number of 0.70 and a transduction efficiency of 54%, whereas, those in cohorts 4 and 5 were given the cell therapy at 2.0 x 106/kg with an average vector copy number of 0.77 and a transduction efficiency of 49%.


1. FDA grants orphan drug designation to Temferon for treatment of glioblastoma multiforme. News release. Genenta Science. March 2, 2023. Accessed March 8, 2023. https://bit.ly/3ylvR4Q

2. A study evaluating Temferon in patients with glioblastoma & unmethylated MGMT (TEM-GBM). ClinicalTrials.gov. Updated January 20, 2023. Accessed March 8, 2023. https://bit.ly/3ZuEKF6

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