The path to bringing a gene therapy to market in the U.S. just got a little rockier. Having expected to swing for approval in the U.S. on the basis of one pivotal trial, uniQure ($QURE) has now learnt the FDA is demanding another clinical study.
Glybera appeared to have a clear path forward as recently as June. At that time, uniQure said the plan was to initiate a clinical trial in 2016 and use the data in a filing with the FDA. However, the FDA has scuttled the plan by telling uniQure it wants to see data from another clinical trial in the BLA filing. What happens now is unclear. UniQure is still planning to start up a U.S.-based trial early next year to meet the post-approval requirements of its European marketing nod, but is "assessing its options" for pursuing approval from the FDA.
Investors reacted badly to the setback, sending shares in uniQure down by as much as 14% in premarket trading. With a big-ticket collaboration with Bristol-Myers Squibb ($BMY) to its name, uniQure is far from being reliant on Glybera. But the treatment for the genetic disease lipoprotein lipase deficiency (LPLD) is both uniQure's best near-term shot at getting a product on the market and a possible harbinger for what is to come for the gene therapies that are following it down the R&D pipeline.
As the advanced party in the gene therapy field, Amsterdam, the Netherlands-based uniQure is used to setbacks. Glybera won approval in Europe in 2012 but a regulatory request for 6-year follow-up data held back its introduction. The recurring question throughout Glybera's slow crawl through the regulatory machinery has been how to assess the safety and efficacy of a new type of product that treats such a tiny patient population. For the FDA, the answer appears to lie in more data. For uniQure and its €1.1 million ($1.2 million) gene therapy, the upshot is more delays and uncertainty.
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