The EMA has told Amicus Therapeutics it lacks the data to file for conditional approval of its Pompe disease drug in Europe. Amicus needs to gather data on more patients and track the progress of previously treated people before seeking conditional approval, delaying the filing to 2019 or beyond.
New Jersey-based Amicus had hoped the unmet needs of patients with the rare metabolic disease would persuade the EMA to allow it to file for approval on the strength of phase 1/2 data. But, having taken a look at the data, the EMA’s scientific advice group has concluded Amicus must build out the clinical package for recombinant human acid alpha-glucosidase AT-GAA before filing.
Amicus left the regulatory discussions with a checklist of tasks it plans to complete to strengthen its hand in discussions with the EMA next year. By then, Amicus expects to have assessed the effect of moving from enzyme replacement therapy (ERT) to AT-GAA on another 10 patients. Amicus will also have 18-month data on the first 19 patients in the phase 1/2, and results from a natural history study of 100 ERT-treated Pompe patients.
Equipped with those data, Amicus may find the EMA more amenable but the delay and its impact on the prospects of AT-GAA have dented investor confidence. Amicus traded down 10% in aftermarket trading on the back of the update.
The dip reflects the passing of a chance to start making sales in Europe and the possible implications for upcoming talks with the FDA. Amicus is set to meet with the U.S. agency in the third quarter to discuss matters including a pathway toward accelerated approval. The biotech will have more data by then but the EMA’s position raises doubts about whether it will have enough to secure a shortened path to market.
Amicus will also use the meeting with the FDA to get feedback on the design of its upcoming phase 3 trial. Once Amicus has the feedback, it will push toward a targeted 2018 start for a pivotal trial that it hopes will meet the needs of regulatory agencies on both sides of the Atlantic.