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European Commission Approves Metabolic Disorder Gene Therapy

DNA strand (Genome.gov)

(Genome.gov)

The biotechnology company uniQure BV in Amsterdam says the European Commission approved Glybera, its gene therapy for patients with lipoprotein lipase deficiency, a rare metabolic disorder. The approval of Glybera, says uniQure, represents the first gene therapy approved by the European Commission.

Lipoprotein lipase deficiency, also called familial hyperchylomicronemia, is an inherited condition that disrupts the normal breakdown of fats in the body. Symptoms of the condition include pancreatitis or inflammation of the pancreas, abdominal pain, enlargement of the liver and spleen, and yellow skin lesions called eruptive xanthomas.

The disorder affects about 1 in 1 million people worldwide, and is caused by a mutation in the LPL gene that normally produces the instructions for producing an enzyme to break down lipoproteins that carry fat molecules from the intestine into the blood stream. Mutations in the LPL gene prevent the enzyme from breaking down those lipoproteins effectively, causing fatty substances build up in the blood stream.

Gene therapy involves replacing or supplementing damaged or mutated genes with healthy genes. The healthy genes are delivered with organisms called vectors that can enter the gene without harm. The uniQure platform uses vectors engineered from harmless adeno-associated viruses to deliver its genetic treatments, including Glybera. The company says its adeno-associated viral delivery method has been tested in more than 60 clinical trials.

The European Commission approval, known as a Marketing Authorisation, covers all 27 member states of the European Union. As part of the approval, Glybera will be administered only through specified medical centers and with trained doctors. The company says it will also create a patient registry to further understanding of lipoprotein lipase deficiency and the Glybera option.

After Europe, uniQure plans to apply for regulatory approval of Glybera in the U.S., Canada, and other markets.

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