The drug Glybera has become the first approved gene therapy in Europe.
Glybera is a treatment for the rare disorder lipoprotein lipase (LPL) deficiency which leaves sufferers unable to digest fats and can result in life-threatening inflammation of the pancreas. It works by delivering functional copies of the gene encoding LPL into muscle cells via a deactivated virus, enabling the cells to produce the missing enzyme. Patients receive a one-time treatment, administered by injection from a specially-trained physician.
Its official approval for market by the European Commission was essentially a formality, following recommendation by the European Medicines Agency (EMA) in July (see previous news).
Approval comes with some restrictions: it can only be used in the most severe cases, and the drug’s developers – Dutch company UniQure – must track outcomes in patients in patients receiving the treatment. Safety concerns have surrounded gene therapy since the death of a US teenager in 1999 led to suspension of gene therapy trials there for four years, and after the occurrence of cases of leukaemia amongst children taking part in two European trials. Because of their experimental nature, gene therapies have tended so far to be trialled only for life-threatening disorders.
Glybera’s licensing is significant for being the first for a gene therapy within the western world, but does not necessarily signal more to come in the near future: no other such treatments are currently under consideration by European regulators. UniQure have said that they will next apply for approval in the US and Canada.