Novogen's Anisina granted FDA Orphan Drug Designation for neuroblastoma

US-Australian drug discovery company, Novogen Limited, today announced that it has received notification from the U.S. Food and Drug Administration (FDA) that its chemotherapy candidate drug, Anisina, has been granted Orphan Drug Designation for neuroblastoma.

Orphan Drug Designation encourages the development of experimental drugs for clinical indications that do not have a high incidence and has been instigated in a number of territories including the U.S, Europe and Australia.

Orphan Drug Designation can provide the following benefits to a drug developer:

• Eligibility for US government grants to defray clinical trial costs
• Tax incentives for clinical research conducted in the United States
• Waiver of US prescription drug filing fees
• Enhanced marketing rights upon Market authorization.

Anisina was granted Orphan Drug Designation for neuroblastoma under the U.S. Orphan Drug Act following a review by the FDA of a package of pre-clinical data submitted by Novogen.

The data is from preclinical studies which were done 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. The key findings 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. The data from these studies were recently announced and presented at Eighth Annual Cancer Molecular Therapeutics Research Association (CMTRA) meeting in Boston, USA.

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."

The ATM program director, Dr Justine Stehn said, "Given it is our intention to take this drug through to the clinic to treat childhood cancer, the designation affords additional guidance from the FDA in the design of our clinical trial program enhancing the efficiency and innovativeness of Anisina's development."

Researchers have demonstrated 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 and Novogen is now 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. 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.