Gene therapy is on the verge of becoming a mainstream treatment option after European regulators recommended that the European Medicine Agency approve Glybera for treating lipoprotein lipase deficiency.
Lipoprotein lipase deficiency is a rare genetic disorder in which the pancreas swells due to the non production of enzyme responsible for breaking down fat and affects just one in one million. Glybera, manufactured by uniQure of Amsterdam, had been initially rejected by the EMA last year but its Committee for Medicinal Products for Human Use revised the decision after it found that the treatment is effective in a small group of people who suffer from severe or multiple attacks of pancreatitis.
Speaking to The Guardian, former director of the US Food and Drug Administration's office of orphan products development, Tim Cote said that the approval of Glybera could prove to be a major milestone in gene therapy treatments.
"This is a watershed moment. Gene therapy holds incredible promise for people, especially those with rare diseases. This is not an isolated example, this is the beginning of something. It paves the way for the approval of other treatments of this kind", he said.