UniQure announced today that the US authorities (FDA) require the company to collect more data before they can ask for market approval for the gene therapy AMT 130.
Some of you will remember that UniQure announced positive data on the efficacy of the therapy back in September last year. However already in November there were signals from the FDA that they would require more data before they could decide on a market approval.
Today it is confirmed again that they want a phase III trial with a placebo group. uniQure states that they are committed to continue to program and the data so far are promising.
For Europe the situation is slightly different, because the European authorities (EMA) did not want a placebo group in the study, for ethical reasons. So, we have to wait to hear more concrete plans ahead, but this study will continue and hopefully deliver more positive data and sufficient numbers for the regulators to evaluate and potentially approve the therapy.
These news are not surprising, however it does imply more time and more effort from the community, says Astri Arnesen, EHA president: “I sincerely hope that people out there don’t lose hope! The data has not changed – they are positive, but the numbers are too small for regulators to be willing to approve – yet. But I am still very hopeful we will get there as soon as possible.”
