November 2, 2012

$1.6m Gene Therapy Drug, Glybera

A $1.6m gene therapy drug, Glybera, for Lipoprotein Lipase Deficiency, LPLD.

Glybera is likely the most expensive drug today. Gylbera is

  • From uniQure
  • Approved by European Commission
  • Available in Europe in second-half of 2013

The disease is caused due to mutation of the LPL gene which causes a decrease in theLPL enzyme activity. 
LPL enzyme is needed in order to break down large fat-carrying particles that circulate in the blood after each meal. When such particles, called chylomicrons, accumulate in the blood, they may obstruct small blood vessels. Excess chylomicrons result in recurrent and severe acute inflammation of the pancreas, called pancreatitis, the most debilitating complication of LPLD.

Glybera gene therapy aims to fix this disease completely by fixing this gene mutation.

Per uniQure (Jörn Aldag,, +31 20 566 8014) 
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. By helping to normalize the metabolism of fat, Glybera prevents inflammation of the pancreas thereby averting the associated pain and suffering and, if administered early enough, the associated co-morbidities

Tags: most expensive drug, glybera drug, LPL enzyme drug, Lipoprotein Lipase Deficiency, LPLD drug, uniqure drug, pancreatitis drug