Something Had to be the Stepping Stone

Medicine is expensive. Most patients aren’t millionaires. It’s disheartening that treatments which could be so beneficial are out of reach for so many. It’s even more disheartening when their production has stopped just because of a lack of consumers. In reality there’s not a lack of demand, just a lack of accessibility. The goal of course, is that the price of the most expensive medicines will eventually decrease, as is the pattern for the majority of new products on the market. But until it becomes affordable, it shouldn’t come as a surprise that demand for the very first gene therapy drug approved, Glybera, hasn’t been high.

In fact, it’s been far from it.

Since UniQure made history in 2014 by receiving approval from the European Commission to treat Familial Chylomicronemia Syndrome patients with Glybera, one individual has been able to afford the 1.4-million-dollar medicine.






UniQure’s approval to market the drug contractually lasted five years, and is set to expire on October 25, 2017.

A few weeks ago, UniQure officially announced they won’t be pursuing a renewal.

As headlines like- “Pulling the plug on the first gene therapy drug”, “Biotech Firm Pulls Pioneering Gene Therapy Due to No Demand”, “First Gene Therapy Ever Approved Calling it Quits”, and “The World’s Most Expensive Medicine is Being Pulled from the Market” filled your newsfeeds, it’s only natural to feel some discouragement. But, please don’t lose all hope.

The thing is, the only reason UniQure made the choice they did is because of a lack of demand, not because a lack of success. Faults weren’t found in the science, but in the commercial side of the drug’s distribution.

Ricki Lewis, author of “The Forever Fix: Gene Therapy and the Boy Who Saved It”, says this is only a small setback in the development of gene therapy. He wants to reassure patients that its time is coming, just maybe not as quickly as we’d hoped.

The Food and Drug Administration has quite a few gene therapy cases on their deliberation table this year. Here’s to hoping that they use Glybera’s failure not as a deterrent to approve gene therapy, but as evidence to alter marketing strategies.

Let’s be clear, Glybera has not been a failure, the system has. Glybera’s demonstrated success has only grown since its first clinical trial.  And this is a reminder too, that participation in clinical trials, with all the attendant risk involved, also has the benefit of potential free access to the best new therapies being tested.

As UniQure continues their examination of gene therapies for diseases like hemophilia B and Huntington’s disease, and the FDA examines other gene therapy proposals, we can cross our fingers they use Glybera as a precedent to continue to explore this exciting new approach to treating difficult disease.

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