UniQure Launches the West’s First Gene Therapy at $1.38 Million

In Clinical Development, Regulation & Government by Cameron

Dutch firm UniQure finally brings to market their ground breaking lipoprotein lipase deficiency gene therapy in Germany with partner Chiesi

After receiving approval for their Lipoproten Lipase deficiency (LPLD) therapy two years ago, UniQure have gathered together their 6-year follow-on data and along with marketing partner Chiesi have field a pricing dossier with Germany’s Federal joint Committee (G-BA). As the first gene therapy to be approved in either Europe or the US, UniQure’s treatment, Glybera, will no doubt set a pricing precedent for other regulators across the west. Whilst the price is not set in concrete, many developers see Germany as the key to winning approval across the EU, and so the G-AB’s response to Chiesi’s requested $1.38 million (€1.11 million) price tag will be of great import to developers and investors across the globe.

LPLD is an ultra rare disease resulting from patients lacking a protein vital for breaking down fat. The result is a build up of fat in the blood, leading to inflammation  the pancreas and potentially diabetes. This gene therapy works by corrective lipoprotein lipase gene to the patient using an adeno-associated vector via injection. The €1.11 million price tag comes from the expected cost of €53,000 per-vial, with an average patient requiring 21 vials for 42 injections. The first treatments can be expect to take place in 2015,

The big question is whether UniQure and Chiesi can demonstrate the cost effectiveness of Glybra. Chiesi have compared the annual cost to expensive enzyme replacement therapies used to treat many other rare diseases. Whilst cost may be an issue, there is much praise for the quality of life improvements that patients can experience through therapies design to eliminate the disease causing mutation, rather than treatments designed to mitigate symptoms.

Investors will no doubt also be watching these developments closely. UniQure and fellow gene therapy developers BlueBird Bio were each successfully floated this year, grabbing $85.4 million and $116.1 million respectively. The G-BA’s response could well effect the prospects of other gene therapy developers such as Spark Therapeutics, Dimension Therapeutics, and Oxford BioMedica.

See Reuters’ take here.