Nuvilex, a clinical-stage biotechnology company providing cell and gene therapy solutions for the treatment of diseases, has submitted, through its subsidiary Nuvilex Australia, an application to the Therapeutic Goods Administration (TGA) in Australia to obtain the Orphan Drug Designation for its pancreatic cancer treatment.

This submission follows similar applications to the United States Food and Drug Administration and the European Medicines Agency in the European Union (EU). Like those in the US (7 years) and the EU (10 years), granting of the Orphan Drug Designation carries with it a significant period of marketing exclusivity. In Australia this period is 5 years.

Nuvilex's CEO and President, Kenneth L. Waggoner, commented, "The application for the Orphan Drug Designation in Australia is the third such application made to the drug regulatory authorities in major areas of the world. In order for the Orphan Drug Designation to be granted to a particular drug or treatment, the target disease must be considered life-threatening and 'rare.' Pancreatic cancer, no matter where it occurs in the world, is undoubtedly life-threatening and, in our opinion, its prevalence in Australia classifies it as a rare disease. It is expected that this application to the TGA will be reviewed relatively quickly, and we may have a decision on it before the end of the year."

Nuvilex's pancreatic cancer treatment consists of implanting living cells that have been encapsulated in pin-head sized, cellulose-based, protective "cocoons" through the use of the proprietary Cell-in-a-Box(R) technology, followed by the administration of low doses of the anticancer prodrug ifosfamide. Ifosfamide is a drug that must be converted into its "cancer-killing" form for it to be effective. The cells that are encapsulated are efficient and effective at carrying out this conversion. By placing these encapsulated ifosfamide-activating cells in proximity to the pancreatic cancer, the anticancer effects of the drug are optimized. In addition, as shown in early-phase clinical trials, because low doses of the anticancer drug are used the side effects from it are greatly reduced in severity and the treatment is well tolerated by the patients undergoing this novel treatment.

Waggoner continued, "As with the applications for the Orphan Drug Designation in the US and the EU, we are extremely grateful to our colleagues at Clinical Network Services (CNS) for their assistance in the preparation of this Australian submission. In particular, the services of Dr. Natalie Thomas in the preparation and submission of this application were invaluable. CNS, one of Australia's leading Contract Research Organisations, has been contracted by Nuvilex to conduct its phase 2b pancreatic cancer clinical trial in that country."