First gene therapy for Europe moves closer to launch

Germany’s federal joint committee (G-BA) is scrutinising Glybera (alipogene tiparvovec), a treatment for the rare genetic condition lipoprotein lipase deficiency (LPLD), which, if approved, would knock Alexion’s Soliris off the top spot as the most expensive drug.

Glybera has been developed by Dutch biotech uniQure with marketing partner Chiesi and is asking a price equating to €1.1 million (£870,000) for a typical course of treatment for a patient with LPLD. The final price will be set after the German authority gives its verdict on its benefits, set for April 2015, and will be subject to a discount under the country’s drug pricing system. UniQure will receive net royalties of 23-30 per cent on sales.

The drug’s backers insist that it is cost-effective, as it could represent a permanent cure for patients. Additionally, only 150-200 people are likely to be eligible for the treatment in Europe, representing a small impact on healthcare budgets.

There is a gene therapy available in China for head and neck cancer, but Glybera is the first for which approval is being sought in the West. UniQure also plans to apply for marketing rights in the US in due course.

The drug’s arrival at this point has not been easy. UniQure acquired Glybera when it took over biotech Amsterdam Molecular Therapeutics (AMT) in 2012. AMT had submitted Glybera for EU regulatory approval but the company was dissolved before the Committee for Advanced Therapies (CAT) and the Committee for Medicinal Products for Human Use (CHMP) reversed their initial decision to reject the drug in 2012.

UniQure’s partnership with Chiesi in 2013 brought €17 million in upfront payments with an equity investment of €14 million for rights to Glybera in Europe and some countries in Asia and the Americas.

With the EU approval came broader market confidence in the potential for gene therapies. uniQure’s pipeline of adeno-associated virus (AAV)-based gene therapies is initially focusing on orphan diseases, but it is also in partnership with others to target chronic and degenerative diseases affecting larger populations. To this end, it recently acquired the cardiology gene therapy company InoCard, now known as uniQure Germany.

It also has a haemophilia B programme in phase I/II trials and, if successful, the new therapy could enable haemophiliacs to produce factor IX on their own following a one-off treatment. The company says it is on track to dose the first patient in early 2015, with initial results expected in mid-2015.

Gene therapies for more common conditions are likely to cost less as developers should be able to recover their R&D outlay from a larger patient pool.

Big pharma has a close eye on the field, with Bayer making a deal with Dimension Therapeutics in June and Novartis setting up its Cell and Gene Therapies Unit in the summer.

Last month, Spark Therapeutics’ night blindness gene therapy received breakthrough status in the US.


Spark’s gene therapy gets FDA breakthrough status

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