Costly gene therapy approved for commercial use in Europe
A pioneering gene therapy treatment has been cleared and approved for commercial use in Europe.
The European Commission has given market authorisation to Glybera, meaning it can be sold throughout the EU. It is the first treatment of its kind to be approved in Europe.
Glybera is a gene therapy treatment for lipoprotein lipase deficiency, a rare disease that leaves sufferers unable to process fat.
The disease means fat builds up in the blood leading to abdominal pain and life-threatening inflammation of the pancreas (pancreatitis).
The treatment’s manufacturers say it will be available next year.
Gene therapy as a concept is relatively simple, if there is a problem with part of a patient's genetic code then change the code. The field is plagued with problems, however. Patients have developed leukaemia and in one trial in the US a teenager died.
In Europe and the US, the therapies are used only in research labs.
Developed by UniQure, the therapy uses a virus to infect muscle cells with a working copy of the gene responsible for breaking down fats.
UniQure chief executive officer Jorn Aldag said: "The final approval of Glybera from the European Commission marks a major step forward in making gene therapies available not only for lipoprotein lipase deficiency, but also for a large number of rare diseases with a very high unmet medical need."
The company said it would apply for regulatory approval in the US and Canada.
The downside to the treatment is its cost, Glybera will cost around 1.2 million euros ($1.6 million) per patient.
Aldag argued that a high price was justified because gene therapy restored natural body function and did not just offer a short-term fix.