Glybera is a drug meant to fight a very rare disorder: Lipoprotein lipase deficiency ,(LPLD) which prevents patients from metabolizing certain fat enzymes in the blood . It does this by modifying a patient’s DNA, and the European Commission has approved it for public use and it will be on sale in mid-2013. That makes it the first human gene therapy drug to be approved for use in the West (China’s been doing gene therapy since 2003.)
The promise of gene therapy is clear, even if its route to effective medicine has been anything but. LPLD was a good candidate for gene therapy because it is a single-gene disorder. Gene therapy offers the promise of curing inherited disease, but the most notable successes have been with respect to single-gene disorders, which usually correspond with rare diseases.
Glybera is currently seeking US approval. If you are one of the one-in-a-million that suffer from LPLD, you probably have been following Glybera, but if you think it could help you, talk to your doctor.RELATED