Advertise With UsAdvertise on our extensive network of industry websites and newsletters.
Get the PBR newsletterSign up to our free email to get all the latest PBR
news.Introgen Therapeutics, via its Swedish subsidiary Gendux, has filed an application with European regulators asking for orphan drug designation for the use of its investigational therapy Advexin in the treatment of cancer patients with Li-Fraumeni sSyndrome.
Subscribe to our email newsletter
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.