Gendux AB and Introgen Therapeutics, Inc. have reached agreement with the European Medicines Agency (EMEA) Committee to file for Advexin p53 therapy marketing approval under the EMEA's Exceptional Circumstances provisions. The application will be for the use of Advexin p53 therapy for the treatment of Li-Fraumeni Syndrome (LFS).
LFS is a genetic disorder characterized by inherited mutations in the p53 tumour suppressor gene. Due to these mutations, LFS patients suffer from numerous cancers often presenting with tumours at an early age. Exceptional circumstances provisions are designed to facilitate access to needed treatments for certain Orphan Medicinal Products, an Introgen press release stated.
Gendux also announced that Advexin has been confirmed by the European Commission as an Orphan Medicinal Product and has been entered in the European Community register on orphan medical products under the number EC/3/06/404. In a related European regulatory action, last month Advexin was recommended for Orphan Drug Status for the treatment of LFS by the EMEA's Committee on Orphan Medicinal Products. Orphan drug registration in Europe confers a number of regulatory and commercial benefits for the product including access to protocol assistance, reduced regulatory fees and a 10-year period of marketing exclusivity from the date of marketing authorization approval.
Introgen and Gendux previously announced that Advexin p53 therapy is available on a compassionate use basis to qualified LFS cancer patients.
Dr. Max W. Talbott, senior vice president of Worldwide Commercial Development and Regulatory Affairs at both Introgen and Gendux said, "After very positive discussions with the EMEA, we plan to utilize the European regulatory approach of Exceptional Circumstances Approval to file a marketing application for LFS. This special provision in the European rules allows us to seek an expedited registration of ADVEXIN in Europe while at the same time advancing our head and neck registrations both in Europe as well as the United States."
Over the coming months Introgen and Gendux plan to complete the filing of Advexin applications for the European approval of LFS treatment, the European approval for head and neck cancer, and the U.S. approval for head and neck cancer. The compatibility of EU and US filing and submission requirements facilitate the achievement of these regulatory filing objectives. Introgen and Gendux intend to provide periodic updates on regulatory progress with targeted completion of all applications during the second half of 2007.
Dr. Talbott further stated, "European and US authorities are to be commended for providing regulatory pathways that are conducive to the development of novel, breakthrough cancer therapies such as Advexin. We are committed to expediting Advexin's availability in U.S. and European markets. As we strive to progress Advexin's registration over the coming months, we look forward to providing specific updates on European and US regulatory events."
Advexin is a targeted molecular therapy with broad applicability in a wide range of tumour types and clinical settings because it targets one of the most fundamental and common molecular defects, abnormal p53 tumour suppressor function, associated with cancer initiation, progression and treatment resistance. Advexin has demonstrated increased survival and durable locoregional disease control in recurrent head and neck cancer patients. Prognostic biomarkers can identify patients most likely to respond to Advexin. Advexin has demonstrated clinical activity in a number of solid tumour types in multiple phase 1, 2 and 3 clinical trials conducted worldwide. A request for accelerated approval review for Advexin is now pending at the US Food and Drug Administration (FDA). The FDA has selected Advexin as a fast track program for an unmet medical need and has designated Advexin for orphan drug use for recurrent head and neck cancer.
Introgen expects that an interim efficacy analysis plan for its Phase 3 head and neck cancer studies will be agreed to by the FDA before the end of 2006. Additional regulatory milestones anticipated for completion during 2007 include: Completion and submission to FDA of the Phase 3 interim efficacy analysis data for head and neck cancer; Conditional Approval approach for head and neck cancer application agreed to by EMEA; and, Exceptional Circumstance Approval Application filing for Li-Fraumeni Syndrome completed with the EMEA. Introgen also anticipates that during 2007, all of the necessary filings will be completed for head and neck cancer at FDA and EMEA to support review and advisory committee review meetings, if required, by these agencies.