The FDA granted orphan drug status for Anisina under the US Orphan Drug Act following a review by the agency of a package of pre-clinical data from preclinical studies submitted by the company.
The preclinical trials were carried out as part of the Children’s Oncology Drug Alliance (CODA) involving Australian charity, The Kids’ Cancer Project (Sydney), The University of New South Wales (Sydney), The Nationwide Children’s Hospital (Columbus, Ohio) and Novogen.
Data from these studies showed that Anisina significantly improved the effectiveness of the standard of care microtubule targeting compound, vincristine, in an animal model of neuroblastoma.
Novogen interim chairperson Ian Phillips said: "Obtaining the FDA approved Orphan drug designation for Anisina is an important strategic achievement for us. It demonstrates the company’s commitment to bringing a drug to market which we hope will improve the outcome for children with neuroblastoma.
"These incentives provided by the FDA have the potential to offset those significant costs associated with the clinical development of Anisina."
Researchers have showed efficacy in an animal model of neuroblastoma both as a monotherapy and in combination with vincristine as stated in the company’s announcement earlier this month.
Currently, the company is conducting pre-clinical studies to further validate the combinatorial effect of Anisina with a range of microtubule-targeting compounds in animal models of adult cancer.
Following completion of the company’s 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 US to follow in early 2017.