Amicus Therapeutics (FOLD) Announces Publication of Migalastat Pivotal Phase 3 Data in Fabry Disease

November 11, 2016 7:31 AM EST
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Amicus Therapeutics (Nasdaq: FOLD), a global biotechnology company at the forefront of rare and orphan diseases, today announced that data from the pivotal Phase 3 Study 012 (ATTRACT) evaluating the efficacy and safety of the oral pharmacological chaperone migalastat compared with Enzyme Replacement Therapy (ERT) in individuals with Fabry disease were published online in the Journal of Medical Genetics1.

"We are honored that the Journal of Medical Genetics has published our pivotal Phase 3 ATTRACT study in patients with Fabry disease who were switched from ERT to migalastat,” said Jay Barth, MD, Chief Medical Officer of Amicus Therapeutics, Inc. “We believe that this 18-month, randomized, active-controlled study generated a strong clinical data set for our precision medicine migalastat. This Phase 3 study, together with our Phase 3 Study 011 in Fabry patients who were naïve to treatment, supported the European Commission’s full approval for migalastat in the European Union as a first line therapy for Fabry disease in patients 16 years and older who have an amenable genetic mutation. This publication is a major accomplishment, and I would like to thank the physicians who were investigators in this study, as well as the patients and families who participated in the study.”

Prof. Ulla Feldt-Rasmussen, Chief of Medical Endocrinology, Copenhagen University Hospital stated, “I have been treating patients with Fabry disease for 16 years, and I believe that significant unmet needs remain. Migalastat is an oral, first-in-class precision medicine with a unique mechanism of action that is based on a patient’s genotype. As a lead author of the journal publication, as well as a principal investigator in Denmark for the migalastat clinical studies for seven years, I believe that migalastat is poised to become an important, differentiated treatment option for patients with Fabry disease who have an amenable mutation.”

Migalastat is designed to selectively and reversibly bind with high affinity to the active sites of certain mutant forms of alpha-Gal A, the genotypes of which are referred to as amenable mutations. Amicus commenced the commercial launch on May 31, 2016 following the European Commission’s full approval for migalastat, under the trade name Galafold™, as a first line therapy for long-term treatment of adults and adolescents aged 16 years and older with a confirmed diagnosis of Fabry disease (alpha-galactosidase A deficiency) and who have an amenable mutation.

About ATTRACT (Study 012)

Study 012 was a Phase 3 open-label study that compared oral migalastat to standard-of-care Enzyme Replacement Therapies (ERTs) for Fabry disease (agalsidase alfa or beta). The study enrolled 60 patients (26 males and 34 females) with Fabry disease with amenable mutations in a clinical trial assay who had been treated with ERT for a minimum of 12 months prior to study entry. These patients were randomized 1.5:1 to switch to migalastat (36 patients) or remain on ERT (24 patients) for the primary 18-month treatment period. The co-primary outcome measures were the mean annualized changes in estimated glomerular filtration rate (eGFR) and measured (iohexol) GFR (mGFR) assessed by descriptive comparisons of migalastat and ERT over 18 months. Secondary outcome measures included left ventricular mass index (LVMi), as well as a composite of Fabry-associated clinical events (i.e. renal, cardiac, or cerebrovascular). Upon completion, patients were eligible to roll over into a separate extension to continue migalastat.



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