UniQure drops plans to seek FDA approval for Glybera

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UniQure have abandoned their ambition to win FDA approval for their gene therapy, Glybera. The decision to no longer seek approval to market their $1.2 million-per-course gene therapy in the USA came after the FDA made the request to see data from two clinical trials of the lipoprotein lipase deficiency (LPLD) therapy.

As the product has uncertain commercial prospects, with LPLD affecting 1 in a million people, uniQure won’t be bringing Glybera to market in the USA. Having already gone through clinical trials and regulatory approval in Europe, the move will not harm the commercial prospects of uniQure as Glybera was the first commercially available gene therapy in history.

“We do need to face the reality that the disease that Glybera treats…is extremely rare,” said UniQure CEO Jörn Aldag. During a conference call with investors, Aldag stressed that patient identification and reimbursement are major barriers for the uptake of Glybera but should however, “not be extrapolated to other gene therapies”, as this could shed a bad light on the whole sector.

As uniQure’s stock has fallen by more than 40% since the publication of data from a Phase I/II trial in patients with Sanfilippo B syndrome, they now have an opportunity to rectify the situation where they will present data from a Phase I/II trial of their haemophilia B gene therapy at a JPMorgan conference.

Mudith Jayawardena

Source: uniQure Announces Financial Results for Third Quarter 2015 and Provides Update on Product Pipeline and Company Developments; Press Release