In the United States, Orphan Drug designation is granted by the U.S. FDA to therapies or biologics intended to treat, diagnose, or prevent rare conditions affecting fewer than 200,000 individuals nationwide. This designation was developed to incentivize drug development in the rare disease space as, historically, this has been difficult to do. As such, this designation comes with benefits such as fee waivers and seven years of market exclusivity upon drug approval. According to Pharmaceutical Technology, the FDA recently granted Orphan Drug designation to THIO (6-Thio-2’-Deoxyguanosine) for glioblastoma.
Developed by MAIA Biotechnology, THIO is described as:
a potentially first-in-class small molecule that is the only direct telomere targeting agent currently in clinical development. THIO’s in vitro activity has been studied in models of several tumor types with active telomerase, [which] is present in >85% of human cancers and contributes significantly to the proliferation and reproductive immortality of cancer cells.
Orphan Drug designation was granted based on data provided by MAIA Biotechnology which showed that THIO can contribute to rapid tumor cell death and can successfully cross the blood-brain barrier in murine models.
Outside of glioblastoma, MAIA Biotechnology is exploring THIO as a therapeutic option for hepatocellular carcinoma, small cell lung cancer, and advanced non-small cell lung cancer; the latter is being evaluated in a clinical study.
What to Know: Glioblastoma
Glioblastoma is a rare, aggressive brain cancer that may also be referred to as stage IV astrocytoma. Many people diagnosed with glioblastoma show no prior signs of brain cancer. Symptoms often manifest quickly and may include:
- Muscle weakness and/or paralysis
- Changes in mood, behavior, or personality such as anxiety, rage, or impulsivity
- Frequent or recurring headaches
- Nausea and vomiting
- Poor balance and coordination
- Difficulty thinking or speaking
- Vision abnormalities
- Fatigue
- Lethargy
- Seizures
The exact cause of glioblastoma is unknown. Risk factors include having radiation therapy in the past or having certain genetic conditions. Unfortunately, glioblastoma is often difficult to treat. These tumors are capable of developing their own blood supply, allowing them to overcome treatment and continue growing. More research is needed to identify more effective therapies.
