Migalastat regulatory update

Amicus said it does not anticipate submitting an NDA by year end for migalastat to treat Fabry’s disease after FDA requested additional data analysis. The company did not provide a new timeline for the submission. Based on the final minutes from Amicus’ September pre-NDA meeting with FDA and follow-up interactions with the agency, the biotech will evaluate “several U.S. pathways” for the small molecule that enhances alpha galactosidase A activity, which may include generating additional data on migalastat’s effect on gastrointestinal symptoms to seek full approval or accelerated approval. ...