First gene therapy medicine recommended for approval in the EU

BRUSSELS – The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended uniQure BV’s Glybera (alipogene tiparvovec) for authorisation in the European Union. Glybera is intended to treat patients with lipoprotein lipase (LPL) deficiency.

LPL deficiency is an ultra-rare inherited disorder. Patients with the disorder cannot produce enough LPL, an enzyme responsible for breaking down fats, due to a defective gene. Management of the disorder consists of strict reduction of dietary fat to less than 20% of the daily caloric intake. Many patients find management of the disorder to be difficult, and as a consequence experience life-threatening pancreatitis attacks requiring admission to hospital.

The CHMP recommended the granting of the marketing authorisation under ‘exceptional circumstances’.

Glybera is the first gene-therapy medicine to be recommended for authorisation in the European Union. Gene therapy medicines replace defective genes with working copies. Glybera uses an adeno-associated virus vector to add working copies of the LPL gene into muscle cells in order to enable production of the enzyme in the cells.

Uniqure, the company that markets Glybera, will be required to provide data from a registry set up to monitor outcomes in patients treated with Glybera, which the CHMP review as they become available.