The therapy, given approval in Europe, called Glybera, was developed by uniQure, a Dutch company. It treats lipoprotein lipase deficiency (LPLD), a disease that affects only several hundred people in the European Union and a similar number in North America.
Glybera (alipogene tiparvovec), is a gene therapy treatment for patients with LPLD (also called familial hyperchylomicronemia). Patients with LPLD, a very rare, inherited disease, are unable to metabolize the fat particles carried in their blood, which leads to inflammation of the pancreas (pancreatitis), an extremely serious, painful, and potentially lethal condition. The approval makes Glybera the first gene therapy treatment approved by regulatory authorities in the Western world.
As I stated back in July, the reason I am noting this accomplishment in this space, where I normally write about treatments for ocular diseases is, because it brings “legitimacy” to the whole field of regenerative medicine. As readers of this online Journal are aware, my interest is in the field of ophthalmology. As you may be further aware, I am currently tracking twenty one clinical trials involving the use of stem cells (or cell threapy) to treat ophthalmic disorders and sixteen gene therapy clinical trials. Several of these are showing promising results and the above approval will bring increased attention to the whole of this field, including the ophthalmic trials.
The only drawback that I can see, is the potential cost of the above Glybera treatment.
As further noted in Ms. Whalen’s article, “The drug's approval is a huge step forward for the field because it's the first time a genetic medicine has been licensed," said Len Seymour, a professor of gene therapy at Oxford University who is unaffiliated with the drug's development. “It begins to exemplify what genetically coded medicines can do."
“A one-time injection will carry an eye-popping price in the range of £1.25 million ($1.6 million), according to a company spokesman. National insurers in Europe are likely to pay the tab. In some markets, UniQure may collect payment in installments over five years,”
In an interview, UniQure chief executive Jorn Aldag said the high price is justified in part because the company will have a very small market: only about one or two people per million have the extremely rare disease that the drug treats. That amounts to several hundred people in the European Union. Other drugs for such rare disorders, called "orphan diseases," also carry high prices of up to $300,000 a year, a phenomenon that has drawn criticism from some insurers.
The European approval of Glybera gives a fillip to a field that has had "fits and starts" since gene therapy research began several decades ago, said Ellen Feigal, vice president for research and development at the California Institute for Regenerative Medicine (CIRM).
The most marked setback was the death of one teenager in a gene-therapy study in 1999. Later, several children in two separate gene-therapy trials in Europe came down with leukemia. The results prompted the U.S. Food and Drug Administration to place a temporary halt on certain gene-therapy trials. In April 2003, it eased the ban.
Recent results have proved more promising, including a gene therapy that successfully treated children suffering from a genetic illness that causes blindness. (Editors Note: As shown in my writeups – see Update 8, about those trials for Leber’s Congential Amaurosis.
Last year, researchers at University College London and St. Jude Children's Research Hospital in the U.S. successfully used gene therapy to treat six patients from hemophilia B, a blood-clotting disease.
I hope that this is just the first of many approvals in the regenerative medicine field in the near future.
Editors Note: According to a knowledgeable source (Alexey Bersenev, and his Stem Cell Assays blog), this approval is actually the fourth gene therapy approval in the world. There have been two previous approvals in China in the field of oncology, and one approval in Russia for peripheral arterial disease. To read further about these, please follow this link.