- uniQure's Glybera (alipogene tiparvovec) is an injectable gene therapy designed to treat lipoprotein lipase deficiency (LPLD), a rare, genetic metabolic disease.
- Glybera was approved in the E.U. in 2012, becoming the first-ever approved gene therapy in Europe. However, uniQure is at risk of losing approval unless it delivers additional data requested by European regulators.
- The cost of treatment with Glybera is $1.2 million.
Investors were unsettled by the FDA's request and the prospect that uniQure's U.S. approval could be delayed, or even scuttled. As a result the stock was down 14% on the news Thursday. Fortunately, uniQure has some buffer after inking a $1.1 billion deal with Bristol-Myers Squibb (BMS) in April under which BMS will use uniQure's gene therapy platform to develop drugs for cardiovascular disease.
Nontheless, there is some disappointment as Glybera represents uniQure's best near-term opportunity of gaining an FDA approval. Glybera is moving forward with the planned clinical trial for 2016, with the goal of using the data from that trial both as part of its U.S. application, and also to satisfy demands from European regulators.
Gaining approval for gene therapies is a long, arduous, and often futile process, but uniQure is determined to move forward and consolidate its presence as a leader in gene therapy.