First gene therapy approved

Jörn Aldag uniQure CEO Credit: uniQure

The first person to be administered a commercial gene therapy will be treated in Germany in the middle of 2013. The European Commission granted marketing authorization for Glybera (alipogene tiparvovec) for the treatment of the ultra-rare inherited disorder, lipoprotein lipase (LPL) deficiency, on November 2. The approval marks the beginning of a significant growth path for gene therapy, says Jörn Aldag, CEO of Glybera's developer, uniQure BV (formerly Amsterdam Molecular Therapeutics), based in Amsterdam. “The world has been watching very skeptically, questioning if a gene therapy could be approved at all. We have overcome all the barriers and now the European Medicines Agency (EMA) has validated Glybera and said it can be applied in [a] market environment.” One of Europe's leading advocates for rare-disease patients, Alastair Kent, says he is “very pleased with the outcome.” However, Kent, who is director of Genetic Alliance UK in London and president of the European Genetic Alliances' Network located in Amsterdam, cautions that it remains to be seen if this really will open up the gene therapy market. “We'll have to watch and wait, but hopefully EMA will be more confident about assessing risks and balances.” uniQure is in the thick of negotiations with reimbursement bodies, trying to agree on a price for a one-off treatment that has lasting effects. The aim is to get a price comparable to the €250,000 ($318,000)-a-year cost of enzyme replacement therapies for treating other rare metabolic disorders. While waiting on approval, the company has screened 319 patients, identifying 32 with the relevant mutation of the LPL gene. In all, Aldag estimates there are 400–500 patients in Europe, and he says Glybera will be profitable going forward. Now uniQure intends to engage with the US Food and Drug Administration, with the aim of launching Glybera in the US in 2014. More ambitiously still, over the next nine months the company will begin phase 1/2 trials of gene therapies in four other disorders.

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Moran, N. First gene therapy approved. Nat Biotechnol 30, 1153 (2012). https://doi.org/10.1038/nbt1212-1153

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