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.

 

Original Article:

http://www.novogen.com/pdf/150716_Anisina_Receives_Orphan_Drug_Status.pdf