The uniQure experience highlights the commercial challenge

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In 2012, uniQure's GLYBERA (alipogene tiparvovec) became the first gene therapy approved in Europe. Yet this remarkable life-changing gene therapy is no longer commercialised because the costs are simply too high.

The issue highlights the challenge companies face in commercialising gene therapies.

GLYBERA is a one-time gene therapy that treats an ultra-rare genetic fat disorder called lipoprotein lipase deficiency (LPL) - LPL affects just one-in-one million people. When launched, it was priced at US$1 million.

GLYBERA works by introducing copies of natural LPL gene to produce functional LPL enzyme indefinitely.

In October 2017, uniQure decided not to renew GLYBERA's marketing authorisation in Europe - it was not commercially viable because of the tiny eligible patient population.

The company has subsequently released data showing its benefit has now been durable for six years.

GLYBERA was never registered in the US because the FDA wanted a second clinical trial to complement the pivotal study on which the European regulator granted approval.

At the time the company made its decision to cease commercialisation, uniQure CEO Matthew Kapusta said, "GLYBERA's usage has been extremely limited and we do not envision patient demand increasing materially in the years ahead."

The ongoing cost of commercialising GLYBERA was simply considered too high and the company has subsequently redirected their financial resources to therapies for larger patient populations. 

The high cost was driven by the nature of the therapy - 'one-time cure' - with the European regulator requiring the company to monitor patients, conduct a phase 4 clinical trial, undergo annual regulatory inspections and increase risk management precautions.  

uniQure redirected US$2million in annual costs from GLYBERA to advancing its pipeline of experimental treatments for haemophilia, Huntington’s disease and other disorders with larger patient populations.

According to Bloomberg, the company is currently weighing its options for a sale or commercial partnerships with larger companies. UniQure’s shares have risen more than 151 per cent this year, giving it a market capitalisation of around US$2.7 billion.

The fact a 'curative gene therapy' can be withdrawn from the market because of its lack of commercial viability could alarm Australian policy-makers.

Australia is already behind given the absence of gene therapies already approved in the US and Europe.

Most of the one-time gene therapies in the later stages of development treat rare hereditary diseases. It is not unrealistic to consider the costs of bringing them to Australia may not be non-commercial for these small to mid-size biotherapeutic companies.

There is even no certainty gene therapies owned by large global biopharmaceutical companies with a presence in Australia will be commercialised locally given the time, effort and investment required.

Dr Amanda Ruth (