Approval for first gene therapy drug

Alipogene tiparvovec, marketed as Glybera, has become the first gene therapy drug to win approval in either the US or the EU. The drug has been approved in Europe for the treatment of lipoprotein lipase deficiency (LPLD), also called familial hyperchylomicronemia, a very rare, inherited disease, which prevents the metabolism of fat particles in the blood and leads to potentially lethal inflammation of the pancreas as a result of blockages caused by the build up of fat particles. It was developed by UniQure, a drug company based in the Netherlands.

Gene therapy is – broadly speaking – the use of coding DNA to prompt the body to correct or mitigate the behaviour of faulty genes, often by providing a working copy of the gene. Glybera injections contain the human lipoprotein lipase (LPL) gene variant LPLS447X in a vector.