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European Commission stamps approval on first gene therapy for rare disease

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There's important news today from the gene therapy front. UniQure won approval for its gene therapy for an extremely rare condition from the European Commission, making the Netherlands-based biotech's product the first gene therapy approved in the Western World. And the first-of-its-kind regulatory nod provides another sign of recovery for the injured field.

After years of development, uniQure has permission to market its therapy, Glybera, for certain patients with lipoprotein lipase deficiency (LPLD) in the European Union. The patients have an enzyme deficiency that robs them of the ability to metabolize fat-laden particles in their blood, leading to inflammation of the pancreas and other complications such as diabetes. Glybera, which is indicated for patients whose disease can be controlled with diet and suffer from pancreatitis, is designed to mend the genetic defect underpinning the ailment.

One to two patients in a million have the disease, making it an extremely rare condition. Yet patients with the disorder lack treatment options, so Glybera has a unique position in a niche market. UniQure plans to follow up the EC nod for the drug with regulatory submissions in the U.S., Canada and other markets.

"Glybera's approval means LPLD patients, for the first time, have a medical treatment option for a very complex and severe disease," said Prof. John Kastelein of the Department of Vascular Medicine at the Academic Medical Center of the University of Amsterdam, the Netherlands, in a statement. "LPLD leads to acute and recurrent pancreatitis attacks, and in many patients causes early onset diabetes and cardiovascular complications. This therapy will have a dramatic impact on the lives of these patients. Currently their only recourse is to severely restrict the amount of fat they consume."

Regulators had turned down the therapy repeatedly before supporting its approval, Reuters has reported. Those were just mild setbacks in what until recent years was a luckless field: Some patients on experimental gene therapy developed leukemia and one U.S. teen died, the BBC reported. Yet in recent years groups such as uniQure and Cambridge, MA-based Bluebird Bio have generated upbeat results and renewed faith in the field among investors.

Yet there are all kinds of questions to answer about reimbursement. For instance, what's the value of a therapy that could correct a genetic defect for life with a single dose?

- here's uniQure's release
- check out the BBC's article

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