Glybera is the first gene-therapy medicine to be recommended for authorisation in the European Union. Gene therapy medicines have the potential to cure genetic disorders by replacing a defective gene with a working copy, thus helping the body to recover functionality. Glybera uses an adeno-associated virus vector as the delivery vehicle to add working copies of the LPL gene into muscle cells to enable production of the enzyme in the cells.
It is intended to treat lipoprotein lipase (LPL) deficiency in patients with severe or multiple pancreatitis attacks, despite dietary fat restrictions.
LPL deficiency is an ultra-rare inherited disorder estimated to affect no more than one or two people per million. Due to a defective gene, patients with this disorder cannot produce enough LPL, an enzyme responsible for breaking down fats. So far, management of patients with the disorder consists of strict reduction of dietary fat to less than 20% of the daily caloric intake. It is very difficult to comply with such a dietary regimen and as a consequence many patients experience life-threatening pancreatitis attacks requiring admission to hospital.