Gene therapy targeting inherited cancer is world's first

An application to review one of the first known treatments for Li-Fraumeni Syndrome (LFS), an inherited form of cancer, has been approved by the European Medicines Regulatory Authority (EMEA).

ADVEXIN has been developed by Dublin-based company Gendux Molecular to reverse one of the most common cancer genetic defects - abnormal p53 tumour suppressor levels that are inherited in Li-Fraumeni patients.

Normal p53, known as the Guardian of the Genome, suppresses cancer and tumour formation. Without it, cancer patients can lose the ability to naturally combat cancer.

Gendux gained marketing authorisation application (MAA) for the drug and is using the Exceptional Circumstances Approval rules available in Europe to ensure the speedy review of potential breakthrough therapies.

The review will be based on clinical efficacy and safety results from the use of ADVEXIN in LFS and in a wide variety of non-inherited cancers with abnormal p53 tumour suppression, the target of ADVEXIN treatment.

ADVEXIN has already demonstrated clinical activity in a number of solid tumour types in multiple phase 1, 2 and 3 clinical trials conducted worldwide. And is being developed for head and neck cancers after showing an ability to control growth in recurrent head and neck, lung, breast, esophageal, and prostate.

Dr Max Talbott, Gendux Molecular's senior vice president of worldwide regulatory affairs, said: "The acceptance of our ADVEXIN application is a historic moment in the development of gene therapy. If approved, ADVEXIN will be the world's first specific treatment for an inherited cancer syndrome and could represent a totally novel, personalised approach for a broad range of cancers."

Gendux is a subsidiary of US-based Introgen Therapeutics.