Think that the US is falling behind? Or is it just an intelligent waiting move? Sometimes being patient pays off. Being first mover with an uncertain technology has often disadvantages. Recently, the European Medicines Agency recommended the approval of a gene therapy drug, called Glybera, for the treatment of lipoprotein lipase (LPL) deficiency.
Lipoprotein lipase (LPL) deficiency is a genetic disorder affecting 1 to 2 persons per million, caused by mutations in the gene that codes the LPL enzyme. This enzyme hydrolyzes consumed food triglycerides into free fatty acids for metabolism. However, the inefficient breakdown of triglycerides leads to a massive accumulation in the blood, sometimes leading nodules under the skin called xanthomas.
Other symptoms include severe abdominal cramping and acute inflammation of the pancreas (pancreatitis) caused by clumps of fats particles blocking capillary flow. Currently, LPL deficiency patients’ only choice is to highly regulate their dietary fat intake. The drug’s maker, Amsterdam Molecular Therapeutics, ran clinical studies on the drug in both the Netherlands and Canada which showed that the drug performed well among the 27 patients tested.
It is only time it gets approved for the floodgates for gene therapy to be opened. With the average development of a drug of 8 years, being a first mover in this industry has tons of risks, but that might be the cost of healthcare innovation.
Amsterdam Molecular Therapeutics was founded in 1998 and based in Amsterdam, is a leader in the development of human gene based therapies. Using adeno-associated viral (AAV) vectors 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. AMT has a product pipeline with several AAV-based gene therapy products in LPL Deficiency, Hemophilia B, Duchenne Muscular Dystrophy, Acute Intermittent Porphyria and Parkinson’s Disease at different stages of research or development.