Dutch biotech UniQure said that the European Medicines Agency's Committee for Medicinal Products for Human Use has recommended approving marketing authorization of its gene therapy product Glybera as a treatment for a rare, inherited disease where patients are unable to handle fat particles in their blood plasma, which leads to recurring severe abdominal pain and pancreatitis.

The European Commission generally follows the recommendations of the advisory committee.

“We expect final approval from the EC within 3 months after the CHMP decision,” says Jörn Aldag, CEO of UniQure. “After today's positive recommendation, Glybera is poised to become the first in a class of gene therapy products approved in Europe to treat orphan diseases, rare conditions with a very high unmet medical need.” Marketing authorization covers all 27 European Union member states.

The recommendation is a big boost for gene therapy, which has seen several setbacks in the course of its 22 year history. The idea is to replace a defective gene with a one that works. Only one gene therapy product has been approved so far, and that was a treatment for head and neck cancer in China. The approval of Glybera is the first gene therapy approval in Europe or the United States.

The path to its approval has not been easy. European regulators had previously rejected it three times, partly due to the size of the trials. The disease, lipoprotein lipase deficiency, affects about one to two people per million. The three trials on which the committee’s recommendation is based, involved only 27 participants. But the committee decided that the benefits of treatment justify its use in the most-severely affected patients.

“Patients with LPLD are afraid of eating a normal meal because it can lead to acute and extremely painful inflammation of the pancreas, often resulting in a visit to intensive care. Now, for the first time, a treatment exists for these patients that not only reduces this risk of getting severely sick, but also has a multi-year beneficial effect after just a single injection,” says Aldag. “The positive recommendation from the CHMP for Glybera therefore represents a major breakthrough for both LPLD patients and for medicine as a whole. Restoring the body's natural ability to break down fat particles in the blood in order to prevent pancreatitis and excruciating abdominal pain suffered by patients, is what gene therapy is all about: curing disease at the genetic level.”

The gene therapy was originally developed by Amsterdam Molecular Therapeutics, a public company that ran out of money while trying to get it approved. UniCure was formed by its majority shareholders Forbion Capital Partners and Gilde Healthcare to acquire AMT’s gene therapy assets, which it did in April when the company went out of business. UniCure’s product pipeline of gene therapy products in development includes treatments for hemophilia B, acute intermittent porphyria, Parkinson's disease and SanfilippoB.