Patients suffering from a rare disease may soon have access to the first commercial gene therapy—if they can afford the million-dollar cost and travel to Europe.

Developed by uniQure, a small Dutch biotechnology company, the therapy is known as Glybera and is intended to help people with lipoprotein lipase deficiency. The disease, which affects one in a million, results from faulty DNA that prevents the breakdown of fats in the bloodstream, resulting in abdominal pain and life-threatening inflammation of the pancreas.

Glybera, which corrects errors in a person’s genetic code, will be available next year, now that the European Medicines Agency has approved its sale. The treatment will cost about 1.2 million euros ($1.6 million) per patient. The company’s goal is to gain the U.S. Food and Drug Administration’s approval for the drug by 2014.

-Noel Brinkerhoff

To Learn More:

Gene Therapy: Glybera Approved by European Commission (by James Gallagher, BBC News)

Glybera Gains Official EMA Nod as First Gene Therapy (by Nuala Moran, BioWorld)

Europe Approves High-Price Gene Therapy (by Ben Hirschler, Reuters)