- GlaxoSmithKline (NYSE:GSK) submits a Marketing Authorization Application (MAA) with the European Medicines Agency seeking approval to market GSK269273, a gene therapy for the treatment of ultra-rare adenosine deaminase severe combined immune deficiency (ADA-SCID). People with the inherited disorder lack virtually all immune protection from bacteria, viruses and fungi. It occurs in one in 200K - 1M newborns worldwide.
- This is the first attempt by a member of Big Pharma to gain regulatory clearance for a gene therapy.
- Only one gene therapy has been approved to date. The EMA cleared uniQure's Glybera (alipogene tiparvovec) in November 2012 for the treatment of familial lipoprotein lipase deficiency, a rare disorder characterized by excess fat in the blood. It has the notoriety of being the most expensive therapy in the world, costing $1M in Germany.
- Previously: UniQure sets price record with Glybera (Nov. 26, 2014)