Amicus Therapeutics highlights new Fabry program data

US biotech firm Amicus Therapeutics (Nasdaq: FOLD) today announced new positive data analyses for the oral small molecule pharmacological chaperone migalastat HCl for Fabry disease at WORLDSymposium 2017 in San Diego, California, USA.

Jay Barth, chief medical officer of Amicus, stated: “The new analyses highlighted at this year’s WORLDSymposium add to the already significant body of data which demonstrate the multiple benefits of treatment with migalastat in Fabry patients with amenable mutations. Here, the correlation of the reduction in disease substrate with the reduction in diarrhea symptoms provides further evidence that migalastat is having a positive effect on an important gastrointestinal symptom in Fabry. These findings in diarrhea symptoms support our current Fabry regulatory strategy for migalastat in the US, which is based upon improvement in diarrhea in this patient population. Also, we remain on track in Japan with our submission to the Pharmaceuticals and Medical Devices Agency based on completed Phase I and Phase III studies. We also remain committed to providing access to this important medicine to patients in the EU, where it is already approved, in addition to other major geographies.”

To support full approval in the USA, which represents around 25% of the global Fabry market, Amicus plans to confirm the clinical beneficial effects of migalastat in a GI symptom study. The GI study is anticipated to begin in 2017 in approximately 35 Fabry patients who are naïve to treatment and who have an amenable mutation and diarrhea and other GI symptoms. More than 50% of patients with Fabry disease report or show GI signs and symptoms, including diarrhea, abdominal pain, constipation, nausea, and vomiting.