The Food and Drug Administration has granted Orphan Drug status to Anisina for the treatment of patients with neuroblastoma. Anisina, made by Novogen, is a small molecule designed to inhibit Tpm3.1-a structural protein and core component of the skeleton or cytoskeleton of a cancer cell-that impacts the function of the protein, causing collapse of the cytoskeleton and cancer cell death.
The Orphan Drug designation-designed to encourage development of drugs in the diagnosis, prevention, or treatment of a medical condition affecting fewer than 200,000 people in the U.S.-grants a product market exclusivity for a seven-year period if the sponsor complies with certain FDA specifications, as well as tax credits and prescription drug user fee waivers. The designation does not, though, shorten the duration of the regulatory review and approval process.
Preclinical studies showed that Anisina 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 presented at recent Cancer Molecular Therapeutics Research Association meeting in Boston.
Novogen is now conducting preclinical studies to further validate the combinatorial effect of Anisina with a range of microtubule-targeting compounds in animal models of adult cancer. Once the company has completed its pre-clinical toxicology program for Anisina, the drug is expected to enter the clinic for adults in mid-2016 with clinical trials in childhood cancer in Australia and the United States to follow in early 2017.