Gene medicine for pancreatitis caused by lipoprotein lipase deficiency Glybera under review in Europe

Glybera, a new gene therapy-based drug to treat lipoprotein lipase deficient patients, Amsterdam Molecular Therapeutics has been submitted to the European Medicines Agency seeking marketing approval.

Glybera is AMT’s proprietary lead product, for lipoprotein lipase deficient patients, stated Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, after submitting the Marketing Authorisation Application (MAA) for the drug.

Amsterdam Molecular Therapeutics has conducted two clinical studies for lipoprotein lipase deficiency (LPLD) in Europe and Canada and long term follow-up from these is ongoing, as is a further study in Canada.

Glybera has shown a sizeable decrease in the incidence of pancreatitis, or acute inflammation of the pancreas, the most debilitating complication of LPLD.

Glybera has an excellent safety profile, these studies also indicated.

“We are delighted to have submitted the Glybera dossier to EMA. We believe that AMT will be the first to successfully develop a gene therapy for a disease caused by a genetic defect,” stated Jörn Aldag, Chief Executive Officer of AMT.

Glybera also validates AMT’s adeno-associated viral (AAV) vector delivery platform, which can be used to deliver other gene therapy products for other indications, Jörn Aldag added.

The MAA for Glybera is now in the validation stage, and the formal review process is expected to begin later this month.

The MAA for Glybera will be reviewed via the centralised procedure which is the standard route for all advanced therapies.

What is lipoprotein lipase deficiency (LPLD)?

Lipoprotein lipase deficiency (LPLD) is a seriously debilitating, and potentially lethal, orphan disease.

Lipoprotein lipase deficiency (LPLD) disease is caused by mutations in the LPL gene, resulting in highly decreased or absent activity of LPL protein in patients.

This protein is needed in order to break down large fat-carrying particles that circulate in the blood after each meal.

When such particles, called chylomicrons, accumulate in the blood, they may obstruct small blood vessels, which in turn can lead to pancreatitis.

Recurrent pancreatitis in LPLD patients can result in difficult-to-treat diabetes. LPLD is associated with significant morbidity and mortality.

No therapy has been approved so far to treat lipoprotein lipase deficiency (LPLD).

Amsterdam Molecular Therapeutics, based in Amsterdam, is a leader in the development of human gene based therapies.

Using AAV as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate what is probably the first stable and scalable AAV production platform. This safe and efficacious proprietary platform offers a unique manufacturing capability which can be applied to a large number of rare (orphan) diseases that are caused by one faulty gene. Currently, AMT has a product pipeline with several AAV-based gene therapy products in LPLD, Hemophilia B, DMD, Acute Intermittent Porphyria and Parkinson’s Disease at different stages of research or development.