The European Medicines Agency has announced it is beginning a formal review of Amsterdam Molecular Therapeutics’ Glybera, to treat lipoprotein lipase deficiency (LPLD), after the drug cleared the validation stage.

LPLD is a seriously debilitating and potentially lethal orphan disease caused by mutations in the LPL gene, for which no treatment exists.

The disease results in decreased or absent activity of LPL protein in patients, which is needed to break down large fat-carrying particles that circulate in the blood after each meal.

AMT has concluded two clinical studies for LPLD in Europe and Canada, and long-term follow-up from these is ongoing, as is a third clinical study in Canada.

Results of these three studies have shown a sizeable decrease in the incidence of pancreatitis, or acute inflammation of the pancreas, the most debilitating complication of LPLD. In addition, these studies indicate that Glybera has an excellent safety profile.

AMT CEO Jörn Aldag said that approval of the MAA for Glybera would fully validate the company’s gene therapy approach and its adeno-associated viral vector delivery platform.

“We believe this step offers hope to many patients, as gene therapy may become the therapeutic approach of choice for inherited disorders,” Aldag said.