Azeliragon for Glioblastoma Earns Orphan Drug Designation


Have you ever heard of Orphan Drug designation? This designation is granted by the U.S. Food and Drug Administration (FDA) to drugs or biologics intended to diagnose, prevent, or treat rare conditions. Rare, in the United States, refers to any condition affecting fewer than 200,000 Americans. As a benefit, drug developers earn incentives such as fee waivers, tax credits, and seven years of market exclusivity upon approval. OncLive reported that azeliragon recently earned Orphan Drug designation for the treatment of glioblastoma. Given that there are so few effective treatment options for glioblastoma, azeliragon has the potential to significantly impact the treatment field if found to be beneficial. 

Canex Pharmaceuticals (“Canex”) licensed azeliragon from vTv Therapeutics, Inc. Prior to being developed for glioblastoma, the drug was being developed as a potential therapy for Alzheimer’s disease. The ALZForum describes azeliragon as:

an oral, small-molecule inhibitor of RAGE, a cell-surface receptor of the immunoglobulin superfamily. Expressed on multiple cell types, RAGE binds advanced glycation end products (AGEs)…[that] when bound to their receptors…cause inflammation and oxidative damage.

Canex hopes that this once-daily therapy, which will inhibit the way RAGE interacts with certain ligands, will ultimately stop glioblastoma from growing or spreading. Glioblastoma tumors are also notoriously resistant to treatment, so azeliragon has the potential to reduce this treatment resistance. Studies so far have found azeliragon to be safe, well-tolerated, and neuroprotective. 

A Brief Overview of Glioblastoma

Glioblastoma is a rare and aggressive form of astrocytoma; it forms in the brain from star-shaped astrocyte cells. This cancer is aggressive because tumors are able to make their own blood supply. Pre-existing genetic disorders, previous radiation therapy, and being male all increase the risk of developing glioblastoma. Symptoms can, but do not always, include:

  • Nausea and vomiting
  • Constant headaches
  • Sleepiness and/or fatigue
  • Weakness on one side of the body 
  • Double or blurred vision
  • Changes in mood, behavior, or personality
  • Difficulty thinking or speaking
  • Seizures

Current treatment options include chemotherapy, electric-field therapy, radiation, and surgery. These treatments are often used in combination to offer the best outcome for patients but are not particularly effective in prolonging survival.

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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