Glybera is the first gene therapy approved by the European Commission in 2012 as a treatment for familial lipoprotein lipase deficiency (LPLD).
Lipoprotein Lipase Deficiency (LPLD) is a rare genetic disorder caused by a mutation in the gene encoding the lipoprotein lipase, an enzyme with an important function in the fat metabolism. People affected by the disease accumulate triglycerides in the blood, due to the loss of lipoprotein lipase function and the resulting inability to clear the fat from the blood. The disease affects one person in a million and often presents in childhood with severe recurring abdominal pain, fat-filled spots known as eruptive xanthoma and attacks of acute pancreatitis, which can cause life-threatening complications for the patients.
Glybera is the first gene therapy approved by the European Commission in 2012 as a treatment for adult patients diagnosed with familial lipoprotein lipase deficiency (LPLD), confirmed by genetic testing, and suffering from severe or multiple pancreatitis attacks despite dietary fat restrictions. It is designed to restore the missing enzyme activity required to enable the processing, or clearance, of fatty acids after a fat-containing meal. Clinicians administer Glybera in a one-time series of up to 60 intramuscular injections in the legs. The patient is administered spinal anesthesia or deep sedation during the procedure. In addition, an immunosuppressive regimen is recommended from three days prior to and for 12 weeks following Glybera administration.
Our clinical development program for Glybera consisted of three noncontrolled, prospective, open-label clinical trials in which we administered Glybera to a total of 27 LPLD patients. In addition, following completion of the clinical trials, we carried out a retrospective case note review of 17 of the 27 patients to determine the impact of Glybera treatment on the frequency and severity of pancreatitis events.
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