Introgen submits MAA for Advexin

USA-based Introgen Therapeutics and its Ireland-based subsidiary, Gendux Molecular, have submitted a Marketing Authorization Application to the European Medicines Agency (EMEA) for Advexin, a targeted p53 tumor suppressor therapy for the treatment of Li-Fraumeni Syndrome cancers.

LFS is a cancer syndrome characterized by the inheritance of an abnormal p53 tumor suppressor. Normal p53 suppresses cancer and tumor formation. Without p53 tumor suppression, LFS patients often develop spontaneous and widespread tumors at an early age. Often, multiple members of the same family suffer from this genetic disorder and there are no approved therapies to specifically treat LFS, Introgen noted.

The company submitted the MAA under the EMEA's Exceptional Circumstances Approval rules for breakthrough therapies for rare diseases such as LFS. Therefore, the drug's potential approval will be based on clinical results from the use of Advexin in LFS, a designated orphan indication for the drug in Europe, and also from results of other trials with Advexin in a wide variety of non-inherited solid tumors that share the p53 biomarker abnormality, which characterizes LFS.