'Seminal moment' for Amicus' Fabry disease drug sends stock soaring

12 July 2017

The US Food and Drug Administration (FDA) has changed its stance on a Fabry disease drug being developed by US biotech Amicus Therapeutics (Nasdaq: FOLD), paving the way for the company to submit a New Drug Application (NDA) later in 2017.

Amicus now plans to submit the NDA for the oral precision medicine migalastat in the fourth quarter of the year, and it is preparing the submission under Subpart H, which provides for accelerated approval.

An additional Phase III study previously requested by the FDA to assess gastrointestinal (GI) symptoms is no longer required prior to the NDA submission, the agency has said after a series of discussions with Amicus.

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