Glybera is the trade name of a medical treatment called alipogene tiparvovec, which was created by UniQure NV in the Netherlands for treating patients with a rare and unusual medical condition called lipoprotein lipase deficiency, which tends to be abbreviated as LPLD for ease of use. Although it is far from being the first gene therapy treatment, Glybera is notable in that it is the first gene therapy treatment to be made available on a commercial basis in either Europe or the United States. As a result, it can seem as though it was something straight out of science fiction, though the results make it clear that it is a real solution to a real problem, albeit an expensive one at $1 million per treatment.
What Does Glybera Do?
LPLD is caused by a harmful mutation that makes it impossible for someone to produce the lipoprotein lipase enzyme on their own, which is a serious problem because said enzyme is responsible for breaking down fatty acids in an efficient and effective manner. As a result, people with LPLD can experience an enormous number of potential complications with their health, ranging from recurring abdominal pain to diabetes, acute pancreatitis, and eruptive xanthomas. Before Glybera was created, medical treatments for the medical condition consisted of various ways to manage its symptoms, ranging from cutting down on the intake of fats and eliminating the intake of simple carbohydrates altogether to both the use of statins for lowering cholesterol and the use of omega-3 fatty acids for lowering lipids. This was because LPLD had no cure, meaning that people with LPLD had to maintain a regular routine of medical treatments.
In contrast, Glybera uses the adeno-associated virus to insert the lipoprotein lipase gene into the muscle cells, thus enabling the production of the lipoprotein lipase enzyme. Since the adeno-associated virus comes with fewer of the side effects that come with some of the other options out there, Glybera can be considered a safe solution to the problem that is LPLD, though it is interesting to note that the medical treatment still comes with the usual immunosuppressive therapies needed to ensure that the immune system will not attack the foreign substances that it has detected.
Why Is Glybera So Expensive?
There are a number of reasons why Glybera is so expensive:
First, while gene therapy treatments have been seeing use since the 1990s, they are still relatively untested combinations of the latest medical techniques and technologies. As a result, they have a commensurate increase in their production costs, which are passed on to the consumers. Furthermore, it is worth noting that Glybera is not a simple and straightforward pill that can be ingested for it to start having its intended effect. Instead, its use is a challenging procedure in its own right, meaning that it needs skilled and experienced specialists needed to get the job done right. Since such skilled and experienced specialists are not available in large numbers, this means a further increase in Glybera’s cost because of the need to compensate said individuals for their limited time and effort.
Second, there are not a lot of people with LPLD in either Europe or the United States or the rest of the world, which means that there is an extremely small market for Glybera, so much so that European people with LPDL can be numbered in the low hundreds. If UniQure NV wants to make back the money that it has sunk into researching and developing the gene therapy treatment as well as pushing it through the approval process in Europe, it needs to set a price that will make it profitable. Unsurprisingly, this is something that Glybera shares with other medical treatments for other rare and unusual medical conditions since the same economic factors exist in those cases as well. Simply put, while it is unfortunate, the for-profit motives of many of the organizations coming up with new solutions to rare and unusual medical conditions means that the smaller number of potential customers must be prepared to pay more.
Finally, it is worth noting that Glybera is meant to last for a long period of time, which is unlike most medical treatments for rare and unusual medical conditions that have to be taken on a regular basis. At the moment, the exact length of Glybera’s usefulness for people with LPLD remains unknown because the studies were conducted within relatively recent years. However, studies involving similar medical treatments using similar methods suggest that the results might last no more than a short number of years, which may or may not be the case with Glybera as well. However, even if Glybera will not last for more than a short number of years, this means that its $1 million should be divided by that number in order to produce an annual figure, which would still make it expensive but not so much so that it would still seem extraordinary among other cutting-edge medical treatments for rare and unusual medical conditions.
With that said, it is important to note that people with LPLD are small in number, meaning that studies of Glybera have been conducted on small sample sizes that would not have been accepted if the circumstances were not so rare and unusual. For example, the first main trial involved no more than 14 subjects, which is why so much still remains uncertain about the medical treatment as well as its effects on people with LPLD.
With that said, it is important to note that Glybera’s $1 million price tag is not necessarily reflective of the price tags of future gene therapy treatments, which will benefit from an increased base of expertise and experience for all sides in the medical treatment development process, much of which will have come from the case of Glybera. As a result, those who are fascinated by the potential of gene therapy treatments should not let this particular case deter them. If anything, it should be seen as an interesting example of the incredible possibilities that have opened up now that such treatments have finally begun to reach the commercial market.