Europe could be months away from having its first approved gene therapy. The Dutch biotech uniQure won a positive opinion from the EMA's Committee for Medicinal Products for Human Use (CHMP) for its gene therapy to treat severe cases of the extremely rare disorder lipoprotein lipase deficiency (LPLD), making the first such regulatory backing for a gene therapy in the Western World.
Once a snake-bitten area of biotech research, gene therapy has regained some credibility as a way to treat genetic disorders. Venture-backed uniQure aims to make Glybera, a gene therapy for patients with defective LPL genes, the first approved treatment of its kind. The European Commission, which often follows CHMP recommendations, is expected to rule on uniQure's application to market Glybera in the EU in the next three months. The CHMP nod was based on evidence from trials of only 27 patients with LPLD, which robs patients of their ability to remove fats from the blood and leads to severe abdominal pain.
"This approval unlocks the potential of gene therapy because it is a first at either the EMA or FDA for gene therapy," uniQure's CEO, Jorn Aldag, said in an interview with veteran Reuters reporter Ben Hirschler. "People have been skeptical as to whether the regulators would buy into this concept, which they have now done."
As Hirschler reports, regulators have passed on uniQure's gene therapy three times before. Investors largely shunned gene therapy after patients on the once-promising genetic treatments suffered sever side effects such as leukemia or, worse, death. Yet the attitudes in the biopharma world have slowly changed because of some successes, including remarkable results from Massachusetts-based bluebird bio's experimental gene therapy in patients with Lorenzo's Oil disease. Sanofi's ($SNY) Genzyme and GlaxoSmithKline ($GSK) have taken an interest in the field, and count on pharma groups to double down on gene therapy if more regulators warm up to the treatments.
Close to a potential EU approval, uniQure plans to seek approvals for Glybera in the U.S. and Canada.