EC Approves Europe's First Gene Therapy

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The European Commission has issued final approval for Europe's first gene therapy-a treatment for a rare genetic disorder that currently has no other treatment options.

The European Commission (EC) has issued final approval for Europe’s first gene therapy-a treatment for a rare genetic disorder that currently has no other treatment options. According to a press statement, the treatment is the first gene therapy to be approved by regulatory authorities in the Western world.

uniQure’s Glybera (alipogene tiparvovec) is intended for patients with lipoprotein lipase deficiency (LPLD) suffering from recurring acute pancreatitis. LPLD is an inherited disorder affecting approximately one or two people per million. Patients are unable to metabolize fat particles in the blood, leading to inflammation of the pancreas and, in some cases, early onset of diabetes and cardiovascular complications.

Using adeno-associated virus vectors as a delivery vehicle, Glybera adds working copies of the lipoprotein lipase gene into muscle cells to enable enzyme production.

“The final approval of Glybera from the EC marks a major step forward in making gene therapies available not only for LPLD but also for a large number of rare disease with a very high unmet medical need,” Jörn Aldag, CEO of uniQure, said in a statement. The company is planning to also apply for regulatory approval in the US, Canada, and other markets. In addition, the company is examining potential treatments for acute intermittent porphyria, Sanfilippo B, hemophilia B, and Parkinson’s disease in other development programs.


The European Medicines Agency (EMA) first recommended Glybera for approval in July 2012, with the EMA’s Committee for Medicinal Products for Human Use (CHMP) recommending the granting of a marketing authorization under “exceptional circumstances.”

uniQure first submitted Glybera as a treatment for LPLD to the EMA in 2009, but received a negative opinion. However, in early 2012, the EC asked the EMA to re-evaluate the application in a restricted group of patients with severe or multiple pancreatitis attacks.“The evaluation of this application has been a very complex process,” said an EMA statement, “but the use of Glybera in a more restricted indication than initially applied for, which targets the patient population with greatest need for treatment, and additional analyses by the Committee for Advanced Therapies have added to the robustness of the data provided and allowed the CHMP to conclude that the benefits of Glybera are greater than its known risks.”

Commercial roll out of the treatment is expected to begin in the second half of 2013. uniQure will be required to provide data from a registry set up to monitor outcomes in patients treated with Glybera, which the EMA will review as they become available.