Last week, the European Medicines Agency the EU analog of our FDA announced that it is, for the first time, recommending a gene therapy product for the treatment of a rare disease called lipoprotein lipase deficiency.
Glybera is the name of the new drug, which replenishes a defective enzyme: lipoprotein lipase (LPL). A deficiency of LPL is caused by the malfunction of the genes that code for this enzyme. Normally, the enzyme breaks down large, fat-carrying molecules that circulate in the blood. However, if LPL is defective, these fat-carrying molecules accumulate, turning the blood milky white and blocking small blood vessels. This, in turn, can lead to pancreatitis.
Glybera therapy, then, counters the deficiency via injection of functional copies of the LPL gene intramuscularly, allowing for the production of active LPL.
If approved, Glybera would become the first gene therapy treatment for disease in the West. Given that the European Commission generally follows the recommendations of the advisory agency, it seems probable that the treatment will become available in all 27 member states of the E.U. by the end of this year.
Small clinical trials conducted at the University of Montreal have shown that the injections are long-lasting and provide partial control of LPL deficiency. Furthermore, Glybera does not appear to have any adverse effects
However, while the approval for Glybera would be a boon for Europe, it remains unclear if or when the company will apply for FDA approval for U.S. marketing.
