With its launch fizzling out, UniQure gives up on $1M+ gene therapy Glybera

UniQure's trailblazing gene therapy has met its end—and its demise offers a lesson to other drugmakers eyeing treatments for ultrarare diseases

After struggling for years to make a commercial success out of Glybera, the world’s first approved gene therapy, uniQure is calling it quits on the ultraexpensive treatment. The drugmaker says it won’t ask European authorities to renew the $1-million-plus gene therapy’s marketing authorization when it expires in October.

The problem? Lack of demand. UniQure CEO Matthew Kapusta said in a release that his company has seen “extremely limited” use of the med since its 2012 approval in Europe. What's worse, uniQure doesn’t “envision patient demand increasing materially in the years ahead,” he added.

Plus, uniQure would have to shell out some cash to win a renewal under the terms of Glybera’s initial approval. The biotech would be required to set up a long-term registry for patient surveillance, complete a postmarketing study, establish new risk-management procedures and undergo yearly reassessments from regulators.

That’s on top of requirements that the company maintain a manufacturing infrastructure and regulatory interactions, all for a med that just wasn’t selling at its astronomical price.

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Last May, MIT Technology Review reported that only one patient had received the drug since its 2012 approval to treat the ultrarare genetic disorder familial lipoprotein lipase deficiency. In order to win coverage, that patient’s doctor had to submit a thick binder of paperwork and personally call a German insurer’s CEO.

All of that worked, and the patient hadn’t been to the hospital once since she was treated, according to the publication. Before Glybera, she had been hospitalized several dozen times.

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Glybera’s demise offers a lesson to other drugmakers working on therapies for super-rare diseases. Despite their ability to greatly benefit patients who have few or no options, the drugs have to be priced within reach.

Speaking with FiercePharma last year, a GlaxoSmithKline spokesperson noted that the pharma giant’s Strimvelis, approved in Europe to treat “bubble boy” syndrome, would be “significantly” cheaper than Glybera. Since then, GSK has placed a $665,000 sticker on the med and offered a money-back guarantee, one potential payment approach for superpricey drugs that put a strain on healthcare budgets.

Chiesi holds European commercialization rights to Glybera, with U.K. Managing Director Tom Delahoyde saying in a statement his company is “extremely disappointed that such an innovative medicine will no longer be available in the country” after the license expires.

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After pulling the plug on Glybera, uniQure will shift its focus to hemophilia B, with plans to push that program into a pivotal trial, Kapusta said. The company also plans to advance its Huntington’s disease candidate into a clinical proof-of-concept study and work on its cardiovascular-focused collaboration with Bristol-Myers Squibb.

In late 2015, after the FDA asked uniQure to conduct new trials on Glybera before approval, the company scrapped plans to continue seeking a U.S. green light.