European regulators have given the green light to UniQure's Glybera, which makes it the first gene therapy to be approved in the western world.
The European Commission has given the green light to Glybera (alipogene tiparvovec) as a treatment for patients with lipoprotein lipase deficiency. LPLD is a very rare, inherited disease, estimated to affect no more than one or two people per million.
Due to a defective gene, sufferers from the disorder are unable to metabolise the fat particles carried in their blood, which leads to potentially lethal inflammation of the pancreas. The approval comes just over three months since the European Medicines Agency's Committee for Medicinal Products for Human Use finally issued a positive opinion on Glybera (see link below).
UniQure chief executive Joern Aldag said that getting final approval "marks a major step forward in making gene therapies available not only for lipoprotein lipase deficiency, but also for a large number of rare diseases with a very high unmet medical need". As part of the approval, the Netherlands-headquartered company noted that patients will receive treatment with Glybera through dedicated centers of excellence and UniQure will also build a patient registry "to further improve the understanding of this devastating, under-researched disease".
Glybera will not come cheap. Administered as a one-time injection, it will reportedly cost £1 million per patient and the company is expected to collect payment Mr Aldag told the Daily Mail that the therapy "provides higher benefit to patients than the classical protein replacement strategy and this is why we think we should be fairly and adequately compensated".
UniQure said it is preparing to apply for regulatory approval in the USA, Canada and other markets. Mr Aldag concluded by saying that the final thumbs-up for Glybera "is an important validation of our innovative product platform and offers strong support for our other advanced development programmes", which focuses on acute intermittent porphyria, Sanfilippo B, haemophilia B and Parkinson’s disease.