The FDA has told Amicus Therapeutics it cannot file for accelerated approval of Pompe disease drug AT-GAA using its existing data. Amicus must now continue adding to its existing phase 1/2 data with a view to sitting down again with the agency next year.
Going into 2018, Amicus harbored hopes that the dire need for new treatments for people with the inherited lysosomal storage disorder Pompe would persuade regulators to wave through AT-GAA on the strength of early-phase data. Those hopes took a big blow in June when the EMA told Amicus it lacked the data for conditional approval in Europe.
Now, the FDA has killed off talk of an application for accelerated approval on the basis of existing data, pushing back a filing until 2019 at the earliest. Amicus is set to meet with the EMA and FDA next year to present its expanded dataset.
Amicus’ plans to resume talks about quickie approvals on both sides of the Atlantic rest on three upcoming data drops. This year, Amicus expects to have 18-month data on 19 participants in its phase 1/2 trial and a readout from its 100-person retrospective natural history study. The final piece will fall into place next year when Amicus shares a look at the progress of 10 patients it enrolled in a later cohort of the phase 1/2 trial.
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Work toward an accelerated approval is advancing in parallel to efforts to generate pivotal data on Amicus’ recombinant human acid alpha-glucosidase. Having spoken to the EMA and FDA, Amicus plans to start a single pivotal trial in 100 Pompe patients by the end of the year. The trial will be open to people who have previously received enzyme replacement therapy and treatment-naïve patients alike.
Amicus will accept patients enrolled in its natural history study into the pivotal program, giving it a readymade pool of people to recruit for the upcoming trial. Participants in the trial will perform the six-minute walk test after 12 months of treatment to generate data on the primary endpoint.