The World’s Most Expensive Medicine Is Being Pulled from the Market

The company that developed the Western world’s first gene therapy will withdraw its pioneering treatment from the market because of a lack of demand.

The drug, Glybera, was the most expensive prescription medication in history at $1 million for a one-time round of injections (see “The World’s Most Expensive Medicine Is a Bust”). It contains engineered viruses carrying copies of a gene meant to correct lipoprotein lipase deficiency, a rare metabolic disease.

Glybera’s approval by European drug regulators in 2012 marked a milestone for the field of gene therapy after a series of setbacks and safety problems. But low demand for the therapy and questions about its effectiveness have plagued Glybera’s manufacturer, UniQure, based in Amsterdam and Lexington, Massachusetts.

“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,” says Casey Quinn, a health economist at the MIT Center for Biomedical Innovation who specializes in European drug pricing.

UniQure said this week that it will not renew the drug’s marketing authorization in Europe when it is scheduled to expire in October. The company will continue to make the drug available to patients until that date.

“The drug’s usage has been extremely limited and we do not envision patient demand increasing materially in the years ahead,” UniQure’s CEO, Matthew Kapusta, said in a company statement.

Quinn says he doesn’t think Glybera’s failure portends trouble for other gene therapies, but the commercial disappointment comes as questions swirl around how future such treatments should be priced.

Several gene-therapy products will be reviewed for approval by U.S. Food and Drug Administration this year, including a treatment for inherited blindness developed by Spark Therapeutics and a novel form of cancer treatment, called CAR-T therapy, being advanced by Novartis and Kite Therapeutics.