Glybera became the first one that gets gene therapy approval

The first gene therapy for rare disease gets approval by European Commission

This is an important news about gene therapy.

After years of development and repeated rejections, uniQure finally got permission to market its gene therapy—Glybera.

Glybera was designed for certain patients with lipoprotein lipase deficiency (LPLD) in the European Union. LPLD is an enzyme deficiency that robs people’s the ability to metabolize fat-laden particles in blood, leading to inflammation of the pancreas and other complications such as diabetes. We say it’s a really rare disease since one or two in a million have the disease. Yet patients with the disorder lack treatment options. So to mend the genetic defect underpinning the ailment seems to be vital.

The approval of Glybera occupies a unique position in a niche market. UniQure now plans to catch regulatory submissions in the U.S., Canada and other markets by following EU nod.

It is the first gene therapy approved in the Western World.