Alipogene tiparvovec (trade name: Glybera) is a gene therapy medicine used to treat lipoprotein lipase deficiency (LPLD). LPLD is an inherited disorder which can lead to very high levels of triglycerides in the blood. Alipogene tiparvovec works by providing the body with a working version of the LPL gene which is normally faulty in people with LPLD. Alipogene tiparvovec is administered by a single injection into the muscle, which is repeated every four weeks for a total of three doses.
Alipogene tiparvovec, sold under the brand name Glybera, is a gene therapy treatment designed to treat lipoprotein lipase deficiency (LPLD), a rare genetic disorder that can cause severe pancreatitis.
It was the first gene therapy treatment to be approved in Europe, in 2012. It works by delivering a healthy copy of the LPL gene, which is responsible for producing lipoprotein lipase, an enzyme that breaks down fats in the blood, into liver cells.
LPLD is caused by mutations in the LPL gene, which prevents the body from producing enough lipoprotein lipase. This can lead to a buildup of fats in the blood, called hypertriglyceridemia, which can cause pancreatitis, a painful inflammation of the pancreas.
Glybera is delivered as a series of injections into the leg muscles. The modified virus in the treatment delivers the healthy LPL gene into the muscle cells, which then begin to produce lipoprotein lipase. This can help to reduce the levels of triglycerides in the blood and prevent pancreatitis attacks.
Studies have shown that Glybera can be effective in reducing the frequency and severity of pancreatitis attacks in people with LPLD. However, it is a very expensive treatment, and it is not available in all countries.