Amicus Pompe Disease Combo’s Approvability In Question As Key Trial Misses Mark

Company Touts ‘Totality Of Data’ As Supporting Approval

Feb 12 2021

• By Alaric DeArment

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The company hopes to score a nod from the US FDA based on a secondary endpoint that won approval for Sanofi’s Lumizyme.

Render illustration of Clinical Trial title on medical documents — Amicus' Phase III study of AT-GAA in Pompe disease failed to meet its primary endpoint, raising questions about the combination's approval prospects. • Source: Shutterstock

Amicus Therapeutics, Inc.’s cipaglucosidase alfa/miglustat for late-onset Pompe disease (LOPD) may be a tough sell for regulators after its pivotal Phase III trial failed to meet its primary endpoint. Mostly – though not entirely – successful numbers on a key secondary endpoint may present a path forward. But even if it does win approval, it could face limited commercial prospects.

Philadelphia-based Amicus announced 11 February results from the Phase III PROPEL study comparing the two-component therapy – known as AT-GAA...

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