FDA clears Amicus to submit migalastat NDA without new trial

Rare disease company Amicus Therapeutics Inc. (NASDAQ:FOLD) gained $2.66 (26%) to $12.92 on Tuesday after it said FDA would no longer require an additional Phase III study of migalastat (AT1001) to treat Fabry's disease, clearing the way for the company to submit an NDA in 4Q17 seeking the compound's accelerated approval. The stock move boosted Amicus' market cap by about $380 million.

In November 2016, Amicus said FDA had determined that reductions in globotriaosylceramide (GL-3), a biomarker used in the company’s Phase III FACETS trial, could not serve as the basis for accelerated approval. To fulfill FDA’s requirements, Amicus planned to conduct a third Phase III trial in about 35 Fabry’s disease patients with amenable mutations (see BioCentury Extra, Nov. 28, 2016)...