First Gene Therapy Approved in the West

The European Medicines Agency took gene therapy a big step closer to normalization this week. The Agency reported on its website that it was recommending approval of the controversial techniques for the treatment of a very rare genetic disorder.

Providing the European Commission take on board this recommendation, and they have always listened to a medical body before, then it will make the first ever regulatory approval of a gene therapy medication, in the West. (A gene therapy medication to treat cancer has approval in China since ‘03). This will mean a metaphorical shot in the arm for a field of study that has for too long labored with too little support and funds.

Gene Therapy from Ceregene

The CEO of Ceregene, a gene therapy company based in San Diego believes an approval such as the European Medicines Agency one, “is really potentially going to change the way the field is looked at, there are no approved products in the major markets, they (big pharma) sell in.” Most pharmaceutical firms have steered clear of investing in gene therapy precisely because of the lack of approvals.

This revolutionary therapy is all about giving the body perfect versions of genes that currently defective and thereby causing health disorders. To date, gene therapy has only been used in clinical trial settings and not in normal medical practice. The European approved gene therapy is one that cure’s lipoprotein lipase deficiency. This is a genetic condition that afflicts just a few hundred patients in the EU and is similarly rare in America.

Glybera from uniQure

Sufferers of this condition have an abnormal genetic code that makes them unable to produce an enzyme that is vital for the breaking down of particular fat-conveying particles that circulate in the bloodstream after consuming food. When these particles are not absorbed, fat builds up to the concentration where blood actually turns white. This leads to periods of acute inflammation of the pancreas, and the only treatment has been a near zero-fat diet. The approved gene therapy is called ‘glybera’ and is мейд by a Dutch company called ‘uniQure’.