WSJ
Gene-Therapy Approval Marks Major Milestone
By JEANNE WHALEN The European Union has approved for sale the Western world's first gene-therapy drug, a type of medicine that introduces functioning genes to patients whose defective genes cause disease.
The costly drug, which treats a rare metabolic disease, is the first gene therapy to be licensed in the U.S. or Europe, though there is a gene therapy for cancer on the market in China, and many others are being tested in clinical trials.
The European approval is a landmark for the field, which is beginning to make progress after years of false starts and high-profile troubles, including the death of at least one patient in a clinical trial in the U.S. in 1999.
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."
The drug, developed by the closely held Dutch company UniQure, is a one-time injection that will carry an eye-popping price in the range of €1.25 million ($1.6 million), a company spokesman said. National insurers in Europe are likely to pay the tab. In some markets, UniQure may collect payment in installments over five years, he said.
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, called Glybera, 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.
Glybera contains a gene that helps the body produce an enzyme that breaks down harmful fats in the blood. Patients with the disease, called lipoprotein lipase deficiency, or LPL, have defective copies of the gene and are unable to process the fat particles, which can lead to potentially lethal inflammation of the pancreas, as well as early onset of diabetes and cardiovascular disease.
The treatment traveled a long road to approval. The European Medicines Agency, or EMA, initially rejected the drug three times, expressing doubts about its efficacy. This time around, the agency change its analysis and approved the use of the drug only in severe cases. The agency finally recommended Glybera for approval in July, a recommendation the European Commission formally endorsed on Friday.
UniQure said it plans to file the drug for regulatory approval next year in the U.S., where there are no gene therapies yet approved for sale.
Investment in gene therapy has boomed in recent years as positive clinical trial results have renewed hopes for the field. The California Institute for Regenerative Medicine, or CIRM, a taxpayer-funded institute that finances cutting-edge medical research, is backing an array of gene therapy studies, including one being conducted by Bluebird Bio of Cambridge, Mass. in a rare disorder called beta-thalassemia.
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 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. 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.
Gene therapy is often attempted in diseases caused by single genes, such as Cystic Fibrosis. Researchers initially hoped that swapping a good gene for a bad would work as a sort of silver bullet in Cystic Fibrosis, but scientists encountered many obstacles, including weak efficacy and side effects, said Uta Griesenbach, a cystic fibrosis researcher at Imperial College London.
Dr. Griesenbach and colleagues this summer began testing a new gene therapy in 130 patients with Cystic Fibrosis—the largest trial yet of a gene therapy for the disease.
Write to Jeanne Whalen at jeanne.whalen@wsj.com
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