The first approved gene therapy product in a Western country

By Professor Juan Tamargo, Madrid, Spain

The agency’s Committee for Medicinal Products for Human Use (CHMP) of The European Medicines Agency (EMA) has for the first time recommended recommended the authorization under “exceptional circumstances” for alipogene tiparvovec, an “orphan medicine” for use in a restricted group of adults and under exceptional circumstances with familial lipoprotein lipase (LPL) deficiency who have severe or multiple pancreatitis attacks despite dietary fat restrictions.

Glybera is made from an adeno-associated viral vector (AAV1) that has been modified so that it carries a gene for LPL but does not replicate. It is injected into a muscle, where it corrects the LPL deficiency by enabling muscle cells to produce LPL.