- UniQure announced quietly on Thursday morning that it would not renew the marketing authorization of its gene therapy Glybera (alipogene tiparvovec) in Europe.
- In October 2012, the European Commission granted the drug a five-year marketing authorization under exceptional circumstances as a treatment for a limited number of patients with of patients with familial lipoprotein lipase deficiency (LPLD).
- The gene therapy was priced at $1.18 million for a course of treatment and the first patient was treated in September 2015. UniQure does not break out revenues from Glybera but the treatment has reportedly only been used once.
After several years of virtually no sales due to its high price tag and small patient population, Glybera has largely been a loss for uniQure. Its abandonment of the asset should come as little surprise to anyone.
The biotech had picked up the rights to the gene therapy in 2012 after its discoverer, Amsterdam Molecular Therapeutics, was forced to liquidate its assets.
Glybera made headlines in 2012 for being the first approved gene therapy, but also for its eye-watering price tag.The drug is administered only once and therefore required a special pricing model. Yet, regulators and pricing authorities were not as open to its million dollar cost, leading uniQure to abandon its pursuit of U.S. approval in 2015.
In order to receive full approval in Europe, uniQure would have had to complete several expensive clinical trials as part of post-marketing agreements, and also would have been required to update its manufacturing in Amsterdam. That plant is now expected to be decommissioned in May.
With hindsight, Glybera could be considered a failed experiment in how to price a gene therapy. Few gene therapies have reached markets, but many more are now approaching regulatory review. With most only requiring a one-time administration, or once annually, pricing remains a hotly debated issue — even without the ongoing tensions over the cost of more traditional therapies. So far, no adequate solution for how to handle this problem has been found, meaning companies entering the space will be forced to see what the market will bear.
UniQure will now turn its focus to a hemophilia B treatment in development. The company previously restructured and had announced it would shed between 20% to 25% of its workforce by the end of 2017.