According to the MIT Technology Review, European gene therapy company UniQure is pulling its services due to lack of customers. Glybera, the company’s formerly available treatment, featured a series of injections to correct the effects of a rare metabolic disease called lipoprotein lipase deficiency. However, the drug’s availability will be limited in the future due to lack of demand for the $1 million treatment.
According to Casey Quinn, a health economist at the MIT Center for Biomedical Innovation, the failure of the drug is attributed to two direct causes; “It wasn’t just that it cost $1 million; it’s that it came to market without much evidence basis that it was worth $1 million,”
The drug is currently sold in Europe, but UniQure will not be seeking renewal on the drug’s marketing authorization. Instead, the company will continue to supply the treatment until the current marketing authorization expires.
Experts predict that this is not the end of gene therapy based medicine; however this report indicates a larger trend in the pharmaceutical and healthcare industry. This issue is central to the ongoing debate in the United States regarding prescription drug pricing; some argue that many prices are predatory, while others assert that they are necessary to absorb high research and development costs. Moving forward, how can companies balance price with research and development costs, effectively enough to provide effective innovative treatments, and remain solvent?
The article notes that additional gene therapy treatments are under approval review by the FDA, including treatments for inherited blindness and a potential application for cancer treatment. Hopefully, these treatments can be offered at a more reasonable price.
Read more at MIT Technology Review