Temferon Granted Orphan Drug Designation for Glioblastoma


Currently, a glioblastoma multiforme (also known simply as glioblastoma or GBM) diagnosis comes with a poor prognosis. There is a 40% survival rate after one year which falls to 17% by the second year. In all, the average person survives for 14-18 months following diagnosis. Current therapeutic options remain largely ineffective for this patient population. Therefore, it is urgent that we discover new, effective, and life-extending treatment options for people with glioblastoma.

According to an article in OncLive, the U.S. Food and Drug Administration (FDA) recently granted Orphan Drug designation to Temferon, a potential treatment for glioblastoma. Orphan Drug designation is granted to drugs or biologics intending to treat, prevent, or diagnose rare conditions (those affecting fewer than 200,000 people nationally). Incentives include fee waivers, tax credits, increased regulatory assistance, and seven years of market exclusivity upon drug approval.

About Temferon

Developed by Genenta Science (“Genenta”), Temferon uses a well-known therapeutic called IFN-α. Previously, the use of this therapeutic fell after IFN-α was found to contribute to systemic toxicity. But Genenta explains

In the tumor microenvironment, IFN-α exerts its anti-tumor activity both directly by promoting cancer cell apoptosis (cancer cell death) and inhibiting vascularization and indirectly by restoring anti-tumor immune response. Temferon modifies TEMs to release IFN-α inside tumors, rebuilding the immune system and counteracting cancer growth.

Genenta is exploring the safety, efficacy, and tolerability of Temferon within the Phase 1/2 TEM-GBM study. Altogether, 21 patients enrolled. Patients are receiving seven different doses and conditioning regimens. Researchers hope to understand hematologic recovery following transplant, toxicity, and levels of differentiated cells.

What is Glioblastoma?

Glioblastoma is a rare, aggressive, and highly malignant form of astrocytoma (cancer that forms from star-shaped astrocyte cells in the brain). While this fast-growing tumor often does not spread throughout the body, it does often invade nearby brain tissue. Glioblastoma tumors can make their own blood supply, allowing them to grow more easily. Unfortunately, this means that these tumors often re-grow following treatment or removal. An estimated 20% of all brain tumors are considered to be glioblastoma. These tumors are more common in males than females. Symptoms relating to glioblastoma can, but do not always, include:

  • Difficulty thinking or speaking
  • Nausea and vomiting
  • Persistent and severe headaches
  • Double or blurred vision
  • Memory loss
  • Weakness on one side of the body
  • Drowsiness
  • Changes in mood, behavior, or personality
  • Seizures

Surgery, radiation, chemotherapy, and electric-field therapy are often used in combination to control or inhibit tumor growth.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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