What Are The Next Steps For Amicus Therapeutics' Migalastat?

Amicus Therapeutics, Inc. (NASDAQ: FOLD) announced the path forward for U.S. regulatory approval for migalastat, including an additional trial in Fabry disease, focused on gastrointestinal (GI) symptoms.

JPMorgan’s Anupama Rama maintains an Overweight rating on the company, while lowering the price target from $12 to $11.

Impact Of Update

Rama expressed disappointment regarding the failure to achieve Subpart H approval for migalastat on a surrogate GL-3/kidney function endpoint.

The analyst believes Amicus Therapeutics shares might decline 15–25 percent following the company’s update, given the current negative sentiment on U.S. migalastat and Zorblisa.

“Looking forward, near-term, our focus will shift to early data from the phase 1/2 Pompe study, which should help establish initial proof-of-concept,” Rama stated.

The Next Steps

The company intends to conduct a randomized, placebo-controlled cross-over 12-month study in about 35 “treatment-naïve” Fabry patients with GI symptoms and amenable mutation, starting in 2017.

“The primary endpoint will assess diarrhea rates based upon established FDA irritable bowel syndrome (IBS) guidance and the data is expected from this study in 2019,” the analyst went on to say.

Although the treatment effect and powering assumptions are not yet known, Rama pointed out that more details would be disclosed closer to the time of the trial's initiation.

At last check, Amicus was down 22.11 percent at $6.48.