Hoping to be the exception

If EMEA approves Introgen Therapeutics Inc.'s MAA for Advexin to treat Li-Fraumeni Syndrome cancers, it would be the first gene therapy to be approved outside of China. The question is how little data the agency will accept for an Orphan disorder for which there are no approved drugs and in which the company conducted no trials.

LFS is an autosomal dominant hereditary disorder involving mutations in the p53 tumor suppressor gene, making carriers highly susceptible to developing a variety of cancers at an early age. Introgen (INGN, Austin, Texas) estimates that LFS affects 800-1,000 families in the U.S. and Europe...