Advaxis Requests Orphan Drug Designation for Treatment of HPV-Associated Anal Cancer with ADXS-HPV

Advaxis Requests Orphan Drug Designation for Treatment of HPV-Associated Anal Cancer with ADXS-HPV

-- Orphan Drug Designation Could Provide Significant Financial Incentives and Serve an Unmet Medical Need--

PRINCETON, N.J.--(BUSINESS WIRE)-- Advaxis, Inc., (OTCQB: ADXS), 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 anal cancer. 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 potentially a seven year market exclusivity period.

"The U.S. Centers for Disease Control and Prevention estimates that 85% of the 7,060 new cases of anal cancer projected for 2013 are caused by HPV. The incidence of anal cancer has increased more than twofold from 1975-2009 and is rising," commented Dr. Robert Petit, Chief Scientific Officer of Advaxis. "Despite treatment with surgery, chemotherapy, and radiation, the 5 year survival for patients with lymph node or distant metastases is less than 50%. We hope that ADXS-HPV will improve survival for patients with HPV-associated anal cancer."

"If granted, Orphan Drug Designation for our lead drug candidate, ADXS-HPV, would 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 waiver of the $1.9 million regulatory application filing fee," commented Thomas A. Moore, Chairman and Chief Executive Officer of Advaxis. "We are looking forward to initial data from our Brown University Study in anal cancer that is currently underway."

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 potentially 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.


ADXS-HPV is an immunotherapy that is designed to target cells expressing the HPV gene E7. Expression of the E7 gene from high-risk HPV variants is responsible for the transformation of infected cells into dysplastic and malignant tissues. Eliminating these cells can eliminate the dysplasia or malignancy. ADXS-HPV is designed to infect antigen-presenting cells and direct them to generate a powerful, cellular immune response to HPV E7. The resulting cytotoxic Tcells infiltrate and attack the tumors while specifically inhibiting tumor Tregs and MDSCs in the tumors that are protecting it.

About Anal Cancer

Anal cancer is a rare malignancy of the gastrointestinal tract, with an estimated 7,060 new cases in the United States in 2013. The most common pathologic type of anal cancer is squamous cell carcinoma (SCCA), which accounts for approximately 85% of all cases. The remaining cases comprise anal adenocarcinoma (10%) and other types (5%). Up to 20% of patients can be asymptomatic at presentation, but most patients present with symptoms related to an anal mass, such as rectal bleeding, perineal pain, the sensation of a mass in the anus, pruritis, and anal discharge. Most patients are diagnosed with localized disease, while approximately 20%-40% of patients present with lymph node involvement, and approximately 10% present with metastatic disease.

Although it is a rare malignancy, the incidence of anal cancer is increasing. From 1973-2000, the incidence of anal cancer in the United States increased by 160% among men and by 78% among women. An estimated 84% of anal carcinomas are caused by HPV; thus, the risk factors for anal cancer are strongly related to sexual practices and behaviors, including number of lifetime sex partners, anal intercourse, and genital warts, all of which reflect an increasing risk of acquiring HPV. The risk of anal cancer is substantially increased in HIV-infected individuals, and an estimated 28% of males with anal cancer during 2001-2005 were HIV-infected.

About Advaxis, Inc.

Advaxis is a clinical-stage biotechnology company developing the next generation of immunotherapies for cancer and infectious diseases. Advaxis immunotherapies are based on a novel platform technology using live, attenuated bacteria that are bio-engineered to secrete an antigen/adjuvant fusion protein(s) that is designed to redirect the powerful immune response all human beings have to the bacteriumto the cancer itself.

ADXS-HPV is currently being evaluated in four clinical trials for human papillomavirus (HPV)-associated cancers: recurrent/refractory cervical cancer (India), locally advanced cervical cancer (GOG/NCI U.S. study, Clinical Identifier NCT01266460), head & neckcancer (CRUK study, Clinical Identifier NCT01598792), and anal cancer (BrUOG study, Clinical Identifier NCT01671488). Advaxis has over 15 distinct immunotherapies in various stages of development, developed directly by Advaxis and through strategic collaborations with recognized centers of excellence such as: the National Cancer Institute, Cancer Research - UK, the Wistar Institute, the University of Pennsylvania, the University of British Columbia, the Karolinska Institutet, and others. For more information please visit: advaxis.comFacebooktwitterLinkedIn

Forward-Looking Statements

This news release contains forward-looking statements, including, but not limited to: statements regarding ADXS-HPV improving survival rates for patients with HPV-associated anal cancer, as well as whether or not ADXS-HPV will obtain Orphan Drug Designation. These forward-looking statements are subject to a number of risks, including the risk factors set forth from time to time in Advaxis' SEC filings, including but not limited to its report on Form 10-K for the fiscal year ended October 31, 2012, which is available at . Advaxis undertakes no obligation to publicly release the result of any revision to these forward-looking statements which may be made to reflect the events or circumstances after the date hereof or to reflect the occurrence of unanticipated events, except as required by law. You are cautioned not to place undue reliance on any forward-looking statements.

Advaxis, Inc.
Diana Moore, 609-452-9814
Director, Investor Relations & Business Development

KEYWORDS: United States North America New Jersey


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