First approved gene therapy sets new price record

1 December 2014

The Western world’s first ever approved gene therapy is set to have a €1.1m price tag when it goes on sale in Germany. 

Dutch biotech firm UniQure and its Italian marketing partner Chiese have set a new record price for a medicine to treat rare diseases with the gene therapeutic treatment Glybera. Besides setting a potential benchmark figure,it also highlights how the conventional pharmaceutical business model may change.

The new gene therapy, Glybera, is a treatment for patients with the rare disease lipoprotein lipase deficiency (LPLD). Patients lack a protein needed to break down fat molecules, resulting in fat build up in the blood, ultimately leading to inflammation of the pancreas and potentially diabetes. Glybera consists of a harmless modified virus that carries a corrective gene into the body’s cells, resulting in the cell regaining sufficient production of the protein for proper function.

Glybera received approval in Europe two years ago but product launch was delayed to allow for the collection of six-year follow-up data on its benefits. UniQure’s success, along with the draw of the high prices that potentially can be charged for some of these rare disease gene therapies, has resulted in a boost for the field; UniQure and BlueBird Bio have both had successful floats on the Nasdaq market in the last 18 months.

Now Chiesi has filed a pricing dossier with Germany’s Federal Joint Committee (G-BA), which will issue an assessment of the drug’s benefits by the end of April 2015. It is seeking €53,000 per vial, which with a total of 21 vials needed for the average patient would equate to a cost of €1.11 million per patient. The price is valid in Germany for one year, but ultimately the figure will be dictated by the outcome of G-BA and negotiations with statutory health insurance funds. According to a spokesperson from Chiesi: " The first commercial treatments are expected in the first half 2015. " UniQure also has plans to seek approval for Glybera in the United States, which it hopes to receive in 2018.

Defending Glybera’s high price-tag, Chiesi said the annualised cost, taking into account the drug’s proven benefits of at least six years, was no more than that charged for some expensive enzyme replacement therapies used in other rare diseases. Despite this, paying for these sky-high gene therapy drugs represents a significant challenge.

UniQure currently has gene therapy programmes for haemophilia and is also looking at gene therapy beyond the rare disease space, with programmes for heart disease and Parkinson’s disease.  

Although Gylbera’s launch price is a benchmark figure, if trials are successful then analysts expect gene therapies for more common conditions to cost less, since there will be a larger patient group for manufacturers to recoup their research and development investments. 

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