First-in-Class ChemoTx Granted Orphan Drug Status for Pediatric Brain CA

VAL-083 previously received its first Orphan Drug designation for the treatment of glioblastoma
VAL-083 previously received its first Orphan Drug designation for the treatment of glioblastoma

DelMar Pharmaceuticals announced that the Food and Drug Administration (FDA) has granted Orphan Drug designation for VAL-083 in the treatment of medulloblastoma, the most common malignant pediatric brain cancer.

In clinical studies, VAL-083 was investigated as monotherapy and in combination with other chemotherapeutic regimens for the treatment of medulloblastoma. The Company's recent pre-clinical research demonstrates that VAL-083 is active against medulloblastoma cells with difficult to treat sonic hedgehog (SHH) characteristics and p53 mutations. Research findings also showed that VAL-083 in combination with temozolomide completely inhibits self-renewal of pediatric brain cancer stem cells (CSCs).

RELATED: Possible New Tx for Pediatric Brain Tumors 

VAL-083 previously received its first Orphan Drug designation for the treatment of glioblastoma. DelMar recently completed the enrollment of a Phase 2 clinical trial in patients with refractory glioblastoma multiforme (GBM). Overall survival data from this trial will be announced in the first half of 2016.

VAL-083 is a first-in-class, small-molecule chemotherapeutic agent.

For more information visit Delmarpharma.com.

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