Medical research has yielded a significant number of breakthroughs that have advanced the field and helped people in need. But in the rare disease sphere, it has sometimes been difficult to incentivize this research. Rare diseases affect fewer than 200,000 people nationwide, which can make it harder to get enough insight about the disease and can also lack profitability for drug developers. This is why the Orphan Drug designation was started: to incentivize drug development in rare disease. Orphan Drug designation comes with benefits such as increased regulatory assistance, fee waivers, and up to seven years of market exclusivity upon the drug’s approval. Pharmaceutical Technology recently shared that ERAS-801 received this designation for the treatment of malignant gliomas such as glioblastoma.
The Urgent Need for Glioblastoma Treatment
Currently, the five-year survival rate for glioblastoma, a rare brain cancer, sits around just 6.8%. This rate has been fairly stagnant over the years. These tumors are capable of making their own blood supply. As a result, glioblastoma is incredibly aggressive. Despite around 100 years passing since glioblastoma was first described in medical literature, there has been little advancement in identifying effective treatment options. The National Brain Tumor Society shares that available treatments are expensive, can damage the brain, and have little effect on improving or lengthening survival rates.
This devastating cancer can cause persistent and severe headaches, difficulty thinking or speaking, nausea and vomiting, double or blurred vision, unilateral weakness, appetite and memory loss, fatigue, seizures, and changes in mood, behavior, or personality. In many cases, glioblastoma can significantly impinge upon quality of life.
ERAS-801: A Potential Treatment Strategy
Outside of Orphan Drug designation, ERAS-801 was also granted Fast Track designation for glioblastoma with EGFR mutations. This oral treatment blocks the EGFR protein to stop the cancer from growing. Preclinical studies highlighted its penetrating abilities in animal models. Now, researchers are exploring its safety, efficacy, and tolerability in the Phase 1 THUNDERBOLT-1 study. More data from the study should be available later this year.
