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Europe's first gene therapy withdrawn

24 April 2017
Appeared in BioNews 897

A million-dollar gene therapy treatment for a rare blood disease will be withdrawn from the European market due to lack of demand.

Gene therapy company UniQure announced on 20 April that it will not pursue the European marketing renewal for its drug Glybera (alipogene tiparvovec) which is due to expire in October 2017. 

'Glybera's usage has been extremely limited and we do not envision patient demand increasing materially in the years ahead,' said Mr Matthew Kapusta, chief executive officer of UniQure, which is headquartered in Amsterdam, The Netherlands. 'The decision to not pursue marketing authorisation renewal of Glybera in Europe involved a thoughtful and careful evaluation of patient needs and the clinical use of the therapy, and is not related to any risk-benefit concern,' he added.

The drug received authorisation in 2012 for the treatment of an ultra-rare blood disorder known as familial lipoprotein lipase deficiency (LPLD) and was the first ever gene therapy treatment licensed in Europe. It launched two years later with a US$1 million price-tag, but has only been used on one patient in Germany since its approval.

LPLD is a genetic condition that affects patients such that their bodies cannot process fat properly. 'You draw blood and you are astonished, there is no red blood, it's cream,' explains Dr Elisabeth Steinhagen-Thiessen, the Berlin physician who treated the only patient who received Glybera. 

That patient appears to be a success story: prior to treatment she was hospitalised over 40 times, but has not had a single admission since. However accessing the treatment was far from easy: Dr Steinhagen-Thiessen faced a huge administrative burden to persuade both the German regulator to permit the treatment, and the patient's insurer to foot the bill.

Ultimately, this may have been the reason Glybera failed. Very small numbers of potential patients, combined with the very high cost have resulted in demand being low.

Renewing authorisation for Glybera for the next five years would come at a price, and there would be significant further costs associated with maintaining a manufacturing infrastructure and associated regulatory reassessments, patient monitoring activities and conducting a post-marketing trial.

Chiesi Farmaceutici in Italy currently holds the European commercialisation rights to Glybera. Mr Tom Delahoyde, the company's managing director in the UK, said in a statement that his company is 'extremely disappointed that such an innovative medicine will no longer be available' after the licence expires.

Firm Pulls World’s First Gene Therapy Treatment: No One Wants It
NBC News |  20 April 2017
First Gene Therapy Ever Approved Calling it Quits
Rare Disease Report |  21 April 2017
The World’s Most Expensive Medicine Is a Bust
MIT Technology Review |  4 May 2016
uniQure Announces It Will Not Seek Marketing Authorization Renewal for Glybera in Europe
GlobeNewswire |  20 April 2017
UniQure Says It Will Not Pursue EC Marketing Renewal for Glybera Gene Therapy
Genetic Engineering and Biotechnology News |  20 April 2017
With its launch fizzling out, UniQure gives up on $1M+ gene therapy Glybera
Fierce Pharma |  20 April 2017
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1 December 2014 - by Arit Udoh 
Glybera, the first gene therapy to go on sale in Europe, is set to cost €1.1m (£870,000) per patient, making it the world's most expensive drug...
11 November 2013 - by Dr Naqash Raja 
The Children's Hospital of Philadelphia has invested US $50 million in a biotech start-up in an effort to commercialise gene therapy...
5 November 2012 - by Dr Lucy Spain 
The European Medicines Agency (EMA) has approved Glybera, a gene therapy to treat a rare genetic condition, lipoprotein lipase deficiency (LPLD), for sale across Europe. Glybera is the first gene therapy to be approved for the Western market and offers the first therapeutic treatment for people with LPLD....
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