Li-Fraumeni syndrome (LFS) is a rare inherited genetic disorder that greatly increases the risk of developing several types of cancer typically with initial occurrence at a young age. The majority of LFS families have inherited mutations in the p53 tumor suppressor gene.
“Cancers resulting from this syndrome are due to abnormal p53 function that is directly addressed by Advexin’s mechanisms of action and is the epitome of targeted molecular therapy,” explained Dr Robert Sobol, Introgen’s senior vice president of clinical and scientific affairs.
Under European Medicines Evaluation Agency (EMEA) rules, treatments for orphan diseases such as LFS may be approved under “exceptional circumstance” provisions. A marketing authorization application filed under these provisions can be reviewed on an expedited and streamlined basis.