FDA grants orphan drug designation to Temferon for glioblastoma multiforme

pharmafile | March 3, 2023 | News story | Medical Communications  

The FDA has granted Temferon an Orphan Drug Designation (ODD) for use as a therapeutic option for patients with glioblastoma multiforme, says clinical-stage immune-oncology company Genenta Science in an announcement.

The safety and efficacy of the drug is being evaluated in the phase 1/2 TEM-GBM trial, which includes up to 21 patients with newly diagnosed glioblastoma with an unmethylated MGMT promoter.

In order to be eligible for the trial, all patients have had to have undergone complete or partial tumour resection, be eligible for radiotherapy, have a life expectancy of at least six months and a Karnofsky performance score of at least 70.

The study’s primary endpoints were Temferon engraftment within the first 90 days, a proportion of patients achieving haematologic recovery 30 days after transplantation and a short-term tolerability of the drug to be shown through stable blood counts, the absence of cytopenias, the absence of significant organ toxicities greater than grade two and the absence of Replication Competent Lentivirus.

Pierluigi Paracchi, chief executive officer of Genenta, commented: “We expect that the FDA’s decision to grant ODD 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 programme. The orphan drug designation programme highlights the significant need for an efficacious therapy for patients suffering with glioblastoma multiforme.”


Betsy Goodfellow

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