DelMar Nabs Orphan Drug Status for VAL-083

The FDA has granted orphan drug designation to DelMar Pharmaceuticals’ VAL-083 for the treatment of medulloblastoma.

Previously, the drug garnered orphan status for glioblastoma in the U.S. and Europe.

Preclinical trial data reveal that VAL-083 targets medulloblastoma cells with sonic hedgehog properties and p53 mutations. Further, in conjunction with temozolomide, it completely thwarts self-renewal of pediatric brain cancer cells.

The company has ongoing clinical trials evaluating VAL-083 for the treatment of glioblastoma multiforme.

Medulloblastoma is the most prevalent form of malignant pediatric brain tumor and accounts for 15 percent to 30 percent of all childhood intracranial neoplasms.