Boost for Gene Therapy Following European Endorsement

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.