Advaxis, a developer of immunotherapies for cancer and infectious diseases, has submitted an application for orphan drug designation with the U.S. Food and Drug Administration (FDA) for ADXS-HPV for the treatment of human papillomavirus (HPV)-associated head and neck cancer.
The orphan drug designation is granted to drug therapies intended to treat diseases or conditions that affect fewer than 200,000 people in the U.S. If granted, it entitles the sponsor to clinical protocol assistance with the FDA, plus federal grants, tax credits, and a seven-year market exclusivity period.
About 50,000 new cases of head and neck cancer are diagnosed annually, with about 15,000 deaths, Advaxis noted in a press release. HPV infection is estimated to account for 20% to 50% of current incidence. More than 80% of new cases occur in men, who are not typically part of HPV vaccination programs, according to the company.
ADXS-HPV is an immunotherapy that is designed to target cells expressing the HPV gene E7. Expression of the 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 T cells infiltrate and attack the tumors while specifically inhibiting tumor regulatory T cells and myeloid-derived suppressor cells in the tumors that are protecting it.
Data from a phase II study in recurrent cervical cancer showed that ADXS-HPV is an active treatment in this HPV-associated cancer, and Advaxis believes ADXS-HPV immunotherapy will show similar activity in HPV-associated head and neck cancer, given the shared causality of the cancers. The company has an ongoing phase I/II study in HPV-positive head and neck cancer in the U.K. and plans to initiate another in the U.S. in 2013.
