1st gene therapy approved in Europe

Health Care, Medicine Comments Off

The European Medicines Agency has for the first time recommended approval of a gene therapy, for the treatment of lipoprotein lipase (LPL) deficiency under “exceptional circumstances”.

The drug alipogene tiparvovec (Glybera, uniQure) was designated an “orphan medicine” on March 8, 2004, and is recommended for use in a restricted group of adults with familial LPL deficiency who have severe or multiple pancreatitis attacks despite dietary fat restrictions.

LPL deficiency is a rare genetic disease that results from a defect in the gene for LPL, an enzyme that breaks down fats. The patient presents with pancreatitis attacks.

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.