Glybera, First Ever Gene Therapy Drug Costs a Whooping $1.4 Million


    

Glybera, the world’s first gene therapy drug goes for a whooping €1.1 million ($1.4 million) sale in Germany, and it is the first pharmaceutical drug with the curative therapies to cure faulty genes.

Manufactured by UniQure, a Dutch biotech firm, and marketed by Chiesi, its Italian marketing partner, Glybera works by replacing defective body genes and malfunctioning cells with corrective genes. It is most effective at treating Lipoprotein Lipase Deficiency (LPLD), an ultra-rare genetic disease that clogs the blood with fat. Glybera consists of modified virus that introduces corrective genes into the cells of patient under treatment.

After 25 years of intensified experiments and several setbacks, Glybera was approved two years ago in Europe, but the manufacturers delayed the launch so that follow-up data on its benefits could be analyzed 6 years after it debuted.

Paying about 1.11 million euros, a typical LPLD patient will need about 42 injections from 42 vials – and the drug works for 6 years. Justifying the high cost of Glybera, Chiesi says the cost is not above what some enzyme replacement therapies cost for some other rare diseases, most especially when weighed against the background that Glybera works for 6 years in any individual.


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