Clinical Trials

Date: 2014-06-03

Type of information: Presentation of results at a congress


Announcement: presentation of results at the 82nd Annual Congress of the European Atherosclerosis Society (EAS) in Madrid

Company: Uniqure (The Netherlands)

Product: Glybera® (alipogene tiparvovec)

Action mechanism:

gene therapy. Glybera® is an adeno-associated viral vector (AAV1) based gene therapy, administered intramuscularly (IM) at multiple-sites in a single session. AAV1 carrying the human variant LPLS447X gene is delivered to skeletal muscle, where it becomes active. The LPL protein is expressed and transported to the capillary endothelium where it binds to chylomicrons and VLDL. Alipogene tiparvovec is intended as a curative measure for patients with LPLD and, as well as enhancing chylomicron metabolism, may prevent episodes of pancreatitis.


lipoprotein lipase deficiency (LPLD)

Therapeutic area: Rare diseases - Genetic diseases


Trial details:

Latest news:

* On June 3, 2014, uniQure, a leader in human gene therapy, announced the full analysis of the six-year follow-up data for patients treated once with Glybera® (alipogene tiparvovec), uniQure’s gene therapy product for the treatment of lipoprotein lipase deficiency (LPLD). Dr. Daniel Gaudet, the leading investigator during the majority of the clinical studies performed with Glybera®, presented the results at the 82nd Annual Congress of the European Atherosclerosis Society (EAS) held in Madrid, Spain. Glybera is approved in the EU as a treatment for LPLD, a potentially life-threatening, orphan metabolic disease that results in patients’ inability to process fat in the blood after a meal. LPLD patients eligible for Glybera® treatment suffer from reoccurring and often severe pancreatitis events leading to recurrent admissions to hospitals and intensive care units (ICUs). The retrospective analysis of hospital records for 19 patients with LPLD compares an equal time period of up to six years before and after treatment with Glybera®. The analysis demonstrates that after a single administration of Glybera, patients have experienced reductions in both the frequency and severity of pancreatitis, long-term clinical benefits that reduce the burden on healthcare resources. 

n =19

Pre-treatment (up to 6 years)

Post-treatment (up to 6 years)


Rate (events/year)

Total hosp. days


Rate (events/year)

Total hosp. days

Documented pancreatitis







Abdominal pain consistent with pancreatitis







Specifically, the data show that these patients have: • a range of approximately 40-50% lower post-treatment documented pancreatitis events and abdominal pain events consistent with pancreatitis;

• no severe pancreatitis up to six years post-treatment; and

•  an approximately 50% lower post-treatment hospitalization rate and number of days spent in the hospital for documented pancreatitis, including only one ICU stay that occurred following treatment.
Glybera was generally well tolerated, with most adverse events such as myalgia, muscular stiffness, and burning sensation from injection site reactions occurring around the time of administration. No long-term safety concerns have been identified.

The aim of the analysis was to measure incidence and severity of acute pancreatitis episodes and utilization of health resources (hospitalizations, ICU stays, days-in-hospital) over an extended period up to six years post-treatment. An independent adjudication board of three experts blinded to patient identification and pre- or post-treatment period information performed a retrospective review of individual hospital records and categorized each abdominal pain event that lead to hospitalization before and after treatment with Glybera. This six-year analysis represents an extension of the three-year retrospective review that formed a part of the data package upon which the European Commission approved Glybera in October 2012 under exceptional circumstances for the treatment of a subset of patients with LPLD. The data confirm the trend exhibited in the first case note review.

Glybera® is indicated for the treatment of adult patients diagnosed with familial LPLD confirmed by genetic testing and suffering from severe or multiple pancreatitis attacks despite dietary fat restrictions. 

Is general: Yes