A treatment for patients who cannot produce enough of an enzyme that is crucial for breaking down fat has become the first gene therapy product to make it onto the market in the western world.
Amsterdam-based biotech company uniQure said today that the European Commission had approved its Glybera (alipogene tiparvovec) product for a subset of those suffering with familial hyperchylomicronemia, also called lipoprotein lipase deficiency (LPLD).
Europe’s drug regulator had already recommended the drug for approval in July and the Commission’s move is largely a rubber stamp. It is, though, a significant moment for gene therapies, which have had trouble moving from bench to bedside (see ‘Europe nears first approval for gene therapy‘).
“The final approval of Glybera from the EC marks a major step forward in making gene therapies available not only for LPLD but also for a large number of rare diseases with a very high unmet medical need,” said Jörn Aldag, CEO of uniQure, in a statement.
But whether it heralds a new dawn for the area is uncertain. Writing for Nature earlier this year, Fulvio Mavilio, the scientific director of Genethon in Evry, France, warned that “Although the approval of Glybera is a positive move, it is unlikely to herald a new age of gene therapies — not without significant changes to the system. It is no coincidence that no gene therapy has yet been approved in the United States and that no other gene-therapy product is being considered by regulators in Europe.”