UPDATE 1-European regulators back first gene therapy drug

Posted: July 20, 2012 at 1:12 pm

* Glybera recommended for lipoprotein lipase deficiency

* First (OTC BB: FSTC.OB - news) gene therapy drug to reach market in West

* Green light follows three previous rejections

* Decision is victory for private Dutch biotech firm uniQure (Adds EMA comment, uniQure investors, more background on gene therapy)

LONDON, July 20 (Reuters) - 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 (Taiwan OTC: 8271.TWO - news) ).

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.

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UPDATE 1-European regulators back first gene therapy drug

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