Glybera, the world’s first drug to treat rare genetic condition lipoprotein lipase deficiency LPLD, is set to go on sale next year in Germany with a €1.1million ($1.4 million) price tag. The gene therapy has been developed by the Dutch biotech firm UniQure that is seeking €53,000 ($66,181) per vial, which equates to $1.4 million for a typical LPLD patient’s treatment, who will need 42 injections from 21 vials.
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. LPLD results in hyper-chylomicronemia, or dramatic and potentially life-threatening increases in the level of large fat-carrying particles, called chylomicrons, in the blood after eating.
In many cases, LPLD and the associated elevated levels of chylomicrons can cause acute and potentially life-threatening inflammation of the pancreas, known as pancreatitis, thus leading to frequent hospitalizations. Recurrent pancreatitis can lead to chronic abdominal pain, pancreatic insufficiency – which is an inability to properly digest food due to a lack of digestive enzymes made by the pancreas, and diabetes. There is no other approved treatment for LPLD.
The gene therapy is designed to restore the LPL enzyme activity required to enable the processing, or clearance, of fat-carrying chylomicron particles formed in the intestine after a fat-containing meal. The product consists of an engineered copy of the human LPL gene packaged with a tissue-specific promoter in a non-replicating AAV1 vector, which has a particular affinity for muscle cells.
European Commission approved Glybera in October 2012 under exceptional circumstances for the treatment of a subset of patients with LPLD. Glybera currently is not approved for use outside of the European Union. UniQure however, plans to seek approval for Glybera in the United States, which it hopes to get in 2018.
UniQure is also working on gene treatments for haemophilia and has an early-stage project in congestive heart failure.
[Via – Yahoo]