- FDA Agreement Reached on Phase 3 Study Design, Primary Endpoint and Histological Methodology -

CRANBURY, N.J., June 22 /PRNewswire-FirstCall/ -- Amicus Therapeutics (Nasdaq: FOLD) today announced it has commenced the U.S. registration Phase 3 trial with its investigational drug, Amigal(TM) (migalastat hydrochloride) for the treatment of Fabry disease. The Company has reached agreement with the U.S. Food and Drug Administration (FDA) on the key protocol design elements of the pivotal trial, including the use of the surrogate primary endpoint of the change in the amount of kidney interstitial capillary GL-3, the substrate that accumulates in the cells of Fabry patients. In addition, the FDA is in agreement that the Company is eligible to seek Accelerated Approval for Amigal according to Subpart H regulations. The Company has begun submitting the Phase 3 protocol to investigational sites worldwide and expects to begin the dosing of subjects in the second half of this year.

John F. Crowley, President and CEO of Amicus stated, 'The start of our Phase 3 trial with Amigal is a major milestone for Amicus and highlights our transition into a late-stage development company.' Crowley continued, 'We are very pleased with the outcome of our interactions with FDA around the design of this pivotal study and are confident we have set the stage for a successful Phase 3 study. We continue to believe that Amigal may be an important treatment option for patients who suffer with Fabry disease and a significant step forward for them and their families.'

Raphael Schiffmann, MD, Director of the Institute of Metabolic Disease, Baylor Health Care System Foundation, commented, 'Having been involved in Fabry research for more than 15 years and considering the Phase 2 data with migalastat, I am pleased that the Phase 3 trial is starting and look forward to continued involvement with this novel approach for the treatment of Fabry disease.'

Amigal U.S. Registration Phase 3 Trial Design

The Phase 3 trial will evaluate the efficacy, safety and pharmacodynamics of Amigal in males and females with Fabry disease. This trial will consist of a six-month double-blind, randomized, placebo-controlled treatment stage and will enroll approximately 60 subjects who are naive to enzyme replacement therapy (ERT) or who have not received ERT for at least six months prior to the start of treatment with Amigal. The Amigal treatment arm dose and regimen will be 150 mg every other day.

The primary endpoint will be the change in the amount of kidney interstitial capillary GL-3 as measured in kidney biopsies using histology. Secondary endpoints will include safety and tolerability, kidney GL-3 as measured in urine, and an assessment of renal function (including glomerular filtration rate (GFR) and 24-hour urine protein).

Additionally, the FDA and Amicus reached agreement on an improved methodology for the histological evaluation of GL-3 in the kidney biopsies.