UniQure abandons ambition to win FDA approval for €1.1M gene therapy

uniQure CEO Joern Aldag

UniQure ($QURE) has dropped plans to seek FDA approval for its €1.1 million ($1.2 million)-per-course gene therapy Glybera. The decision comes three months after FDA told uniQure it would need to see data from two clinical trials of the lipoprotein lipase deficiency (LPLD) therapy before making a decision.

Faced with the need to run two clinical trials to win approval of a product with uncertain commercial prospects--LPLD affects 1 in 1 million people--uniQure has canned its plan bring Glybera to market in the U.S. The move is unlikely to have a major direct effect on the commercial prospects of uniQure. Having gone through a tortuous clinical trial and regulatory process in Europe, Glybera became the first commercially available gene therapy. But while its place in the history books and importance in establishing a pathway and price for gene therapies is assured, Glybera is something of a curio.

UniQure CEO Jörn Aldag sought to play up the unique characteristics of Glybera in the hours after revealing the decision to back out of the U.S. development program. "We do need to face the reality that the disease that Glybera treats...is extremely rare," Aldag told investors on a conference call. Patient identification and reimbursement are major barriers to the uptake of Glybera. "This situation is unique to Glybera. It should not be extrapolated to other gene therapies," Aldag said. In general, investors have seen events that are bad for one gene therapy as being negative for the whole sector.

Some backers of uniQure certainly saw the Glybera news and other aspects of the company's third-quarter results as bad for the business, sending the gene therapy pioneer's stock down 17% on the day of the news. The stock has now fallen by more than 40% since the publication of data from a Phase I/II trial in patients with Sanfilippo B syndrome in September. UniQure has an opportunity to wipe out the decline just before the start of the JPMorgan conference next month, when it will present data from a Phase I/II trial of its hemophilia B gene therapy.

Amsterdam, the Netherlands-based uniQure had previously expected to have data by the end of the year. When the dataset is published, it will only contain results from two of the five patients dosed to date in the study. The two patients, the first to be dosed in the trial, will have generated 12 weeks of results by the time of the JPMorgan conference. Data from the other three trial participants are slated for publication at a medical conference in the first half of 2016.

- read the release