The €1.1M drug Glybera was the first gene therapy on the European market. UniQure wants to sell it in the US too, but the FDA makes it much more difficult than expected.
UniQure’s financial report of the first half of 2015 shows good results, cash and cash equivalents of the company increased to €181.9M, compared with €53.2M at the end of 2014. UniQure owes this growth mostly to a lucrative collaboration with Bristol-Myers Squibb, which until now made payments of about €125M to the Dutch biotech.
However, UniQure faces a setback concerning its gene therapy Glybera that is approved in Europe since 2012 but still waits for an approval on the US market. Furthermore, this will be trickier than the company has previously thought, as the FDA just stated that it wants “more than one additional clinical study to support a BLA filing”.
UniQure did not give up on its intended US-based clinical trial in early 2016. But the additional requirement leads the company to reconsider its plans. In the report, UniQure states that it is “assessing its options for pursuing regulatory approval of Glybera in the US”.
As FierceBiotech points out, this is not the first delay that UniQure has faced during an approval process. A regulatory request for a follow-up study placed Glybera on hold when submitted to European regulatory authorities. The problem is always that the next generation therapy treats the fat metabolism disorder LPLD, which is extremely rare. UniQure must now ponder how it can get access to the US market effectively.