FDA Grants Orphan Drug Designation to Antitropomyosin Drug for Neuroblastoma


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The U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to Novogen Limited’s chemotherapy candidate drug Anisina for neuroblastoma. Anisina is a small molecule belonging to a family of compounds called antitropomyosins. It has been designed to inhibit Tpm3.1, a structural protein that is a core component of the cytoskeleton of cancer cells.

The decision was based on preclinical data demonstrating that the antitropomyosin drug significantly improved the effectiveness of the standard-of-care microtubule targeting compound, vincristine, in an animal model of neuroblastoma. The data from these studies were recently announced and presented at Eighth Annual Cancer Molecular Therapeutics Research Association meeting in Boston.

Researchers have demonstrated efficacy in an animal model of neuroblastoma both as monotherapy and in combination with vincristine, and preclinical studies to further validate the combinatorial effect of the drug with a range of microtubule-targeting compounds are now being conducted in animal models of adult cancer. ■

 



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