European regulators back First Gene Therapy Drug

Updated 11:05 AM Jul 22, 2012
        LONDON – European regulators have recommended approval of the Western world’s first gene therapy drug – after rejecting it on three previous occasions – in a significant advance for the novel medical technology.
More than 20 years since the first experiments with the ground-breaking method for fixing faulty genes, scientists and drug companies are still struggling to apply gene therapy in practice.
Friday’s decision by the European Medicines Agency (EMA) is a win for the drug’s maker, the small Dutch biotech company uniQure, and a potential lifeline for patients with the ultra rare genetic disorder lipoprotein lipase deficiency (LPLD).
It comes too late, however, for investors in the previous listed firm Amsterdam Molecular Therapeutics (AMT).
After the earlier rebuffs for its Glybera medicine, AMT was taken private by newly created uniQure in April because it could no longer fund itself in the public markets.
Patients with LPLD are unable to handle fat particles in their blood plasma and are afraid of eating a normal meal because it can lead to acute inflammation of the pancreas.
The disorder – estimated to affect no more than one or two people per million – can cause acute pancreatitis and death.
Winning approval for Glybera proved particularly challenging because the company was only able to test it on 27 patients in clinical trials, due to the rarity of the condition.
That thin evidence base made the European agency reluctant to approve the drug initially.
But the London-based watchdog said it now accepted there was sufficient benefit to justify a green light for the worst-affected patients, on condition that those receiving the one-off therapy continued to be followed.
“This approval unlocks the potential of gene therapy because it is a first at either the EMA or FDA for gene therapy,” uniQure’s chief executive Jorn Aldag said.
“People have been sceptical as to whether the regulators would buy into this concept, which they have now done.”
The idea of treating disease by replacing a defective gene with a working copy gained credence in 1990 with the success of the world’s first gene therapy clinical tests against a rare condition called severe combined immunodeficiency (SCID).
People with SCID – also known as “bubble boy disease” – cannot cope with infections and usually die in childhood.
The field then suffered a major setback when an Arizona teenager died in a gene therapy experiment in 1999 and two French boys with SCID developed leukaemia in 2002.
In China, Shenzhen SiBiono GeneTech won approval for a gene therapy drug for head and neck cancer in 2003 but no products have been approved until now in Europe or the United States.
More recently, some large pharmaceutical companies have also been exploring gene therapy. GlaxoSmithKline, for example, signed a deal in 2010 with Italian researchers to develop a SCID therapy. REUTERS

Leave a Reply

Fill in your details below or click an icon to log in: Logo

You are commenting using your account. Log Out /  Change )

Google+ photo

You are commenting using your Google+ account. Log Out /  Change )

Twitter picture

You are commenting using your Twitter account. Log Out /  Change )

Facebook photo

You are commenting using your Facebook account. Log Out /  Change )


Connecting to %s