FDA Grants Orphan Drug Designation to Anti‑Tropomyosin Drug for Neuroblastoma
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 anti-tropomyosins. 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 anti-tropomyosin 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 (CMTRA) meeting in Boston.
Researchers have demonstrated efficacy in an animal model of neuroblastoma both as a monotherapy and in combination with vincristine, and Novogen is now conducting preclinical studies to further validate the combinatorial effect of the drug with a range of microtubule-targeting compounds in animal models of adult cancer.
The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.
