Source: www.marketwatch.com
Author: press release

Advaxis, Inc., a leader in developing the next generation of immunotherapies for cancer and infectious diseases, announced that it has submitted an Application for Orphan Drug Designation with the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD) for ADXS-HPV, its lead drug candidate, for the treatment of human papillomavirus (HPV)-associated head and neck cancer. There are about 50,000 new cases of head and neck cancer annually, with about 15,000 deaths. HPV infection is estimated to account for 20-50% of current incidence.

Orphan Drug Designation is granted to drug therapies intended to treat diseases or conditions that affect fewer than 200,000 people in the United States. Orphan Drug Designation entitles the sponsor to clinical protocol assistance with the FDA, as well as federal grants, tax credits, and a seven year market exclusivity period.

“HPV-associated head and neck cancer is growing at an epidemic rate in the United States and other regions throughout the world. According to the U.S. Centers for Disease Control and Prevention (CDC), over 80% of new cases occur in men, who are not typically part of HPV vaccination programs,” commented Dr. Robert Petit, Chief Scientific Officer of Advaxis. “Data from our Phase 2 study in recurrent cervical cancer show that ADXS-HPV is an active treatment in this HPV-associated cancer. We believe that ADXS-HPV immunotherapy will show similar activity in HPV-associated head and neck cancer, given the shared causality of the cancers. We have one ongoing Phase 1/2 study in HPV-positive head and neck cancer in the United Kingdom and plan to initiate another in the United States in 2013. We believe ADXS-HPV could become an important new non-cytotoxic treatment for patients with HPV-associated head and neck cancer.”

“If granted, Orphan Drug Designation (ODD) for our lead drug candidate, ADXS-HPV, could expedite our ability to help the over 10,000 Americans that the American Cancer Society estimates will be newly diagnosed with HPV-associated head and neck cancer in 2013. ODD would also provide seven years of market exclusivity for ADXS-HPV if it is approved by the FDA, tax credits on U.S. clinical trials, eligibility for orphan drug grants, and a waiver of the $1.9 million regulatory application filing fee,” commented Thomas A. Moore, Chairman and CEO of Advaxis. “It is believed that the number of new HPV-caused head and neck cancers in the United States now approaches the number of new cervical cancer cases. This indication, with these incentives, if achieved, could substantially increase the potential value of the ADXS-HPV franchise.”

About Orphan Drug Designation
Under the Orphan Drug Act (ODA), the FDA may grant orphan designation to a drug or biological product intended to treat a rare disease or condition, which is generally a disease or condition that affects fewer than 200,000 individuals in the United States, or more than 200,000 individuals in the United States and for which there is no reasonable expectation that the cost of developing and making a drug or biological product available in the United States for this type of disease or condition will be recovered from sales of the product. The benefits of orphan drug designation can be substantial and include federal grants, tax credits, and a seven year market exclusivity period once the product is approved, provided that the product is first to market.

In order for a sponsor to obtain orphan designation for a drug or biological product, an application must be submitted to OOPD, and the designation approved. The approval of an application for orphan designation is based upon the information submitted by the sponsor. A drug that has obtained orphan designation is said to have “orphan status.” Each designation request must stand on its own merit. Sponsors requesting designation of the same drug for the same indication as a previously designated product must submit their own data in support of their designation request. The approval of an orphan designation request does not alter the standard regulatory requirements and process for obtaining marketing approval. Safety and efficacy of a compound must be established through adequate and well-controlled studies.