Experimental Drug Gains FDA Orphan Drug Designation for Glioblastoma

GDC-0084 is a small molecule inhibitor of phosphatidylinositol 3-kinase with potential antineoplastic activity.

Kazia Therapeutics Limited received orphan drug designation for its investigational new drug, GDC-0084, by the United States Food and Drug Administration (FDA).

GDC-0084 is being developed to treat glioblastoma multiforme (GBM), the most common and most aggressive variation of primary brain cancer.

GBM is characterized by headaches, changes in personality, nausea, and stroke-like symptoms. It can be the result of genetic disorders like neurofibromatosis or prior radiation therapy. According to the Mayo Clinic, the disease is very difficult to treat and current standard of care consists of therapies include those that slow the progression of cancer and alleviate signs and symptoms.

The cancer type is more frequently diagnosed during a neurological exam or via imaging tests. Approximately 130,000 patients are diagnosed worldwide annually, and median survival on best available care is typically 12-15 months.

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