With China being the first country ever to approve gene therapy, Europe is not too far behind.
Gene therapy is the experimental technique in which physicians use one’s gene to treat or prevent inherited diseases.
The European Medicines Agency (EMA) announced its approval of the Glybera (alipogene tiparvovec). Glybera is aimed to treat lipoprotein lipase (LPL) deficiency in patients who are suffering from severe or multiple pancreatitis attacks.
Affecting between one to two people per million, LPL deficiency is an exceedingly rare hereditary disorder. This deficiency is responsible for the lack of production of LPL, making it hard to break down fat in one’s body. Currently the only remedy for LPL deficiency is a strict dietary routine that consists of eating 20 percent less than the normal daily calorie consumption. According to the EMA, for patients it is difficult to conform to their dietary routine, thus leading to deadly pancreatitis attacks.
The way gene therapy works is by replacing a malfunctioning gene with a functioning copy in order to restore functionality. Glybera uses an adeno-associated virus course as the delivery vehicle to add functioning copies of the LPL gene into muscle cells to aid in production of the enzyme in the cells.
UniQure, which is the company responsible for the research and early development of human gene therapy, believes the approval is progression for patients and medicine as a whole.
In addition to gene therapy for LPL deficiency, CEO of UniQure, Jorn Aldag, stated the company hopes to make advancement in other conditions such as Parkinson’s disease.
"We believe that just like antibodies, gene therapy will one day be a mainstay in clinical practice," he told Associated Press.
Monika Benstetter, the spokeswoman for EMA, wants to make clear that the approval is thus far only for patients with the most significant need.
With gene therapy, EMA hopes to not only limit the risk of patients severely getting sick, but also have a multi-year favorable effect after a single dose.