Novogen, a biotechnology company headquartered in Sydney, Australia announced that the U.S. FDA has granted Orphan Drug Designation (ODD) to its chemotherapy candidate drug, Anisina for the treatment of neuroblastoma.   Neuroblastoma is a rare cancer, but is the most common solid tumor outside the brain observed in children under 5 years of age.  Anisina is a small molecule designed to inhibit Tpm3.1, a structural protein, which is a core component of the cytoskeleton of a cancer cell.  It has been shown that Anisina binds to Tpm3.1 and kills a broad range of cancer cell types by collapsing their cytoskeleton.  The ODD was based on pre-clinical data submitted by the company, which demonstrated that Anisina significantly improved the effectiveness of the standard of care microtubule targeting compound, vincristine, in an animal model of neuroblastoma.


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