Amicus Therapeutics, a biopharmaceutical company at the forefront of therapies for rare and orphan diseases, has conducted a successful pre-submission meeting with the European Medicines Agency (EMA) to discuss the registration of migalastat HCl (migalastat) monotherapy for the treatment of Fabry disease.

Migalastat is an oral small molecule pharmacological chaperone in development for Fabry patients with amenable mutations. Amicus has begun preparing a marketing authorization application (MAA) that it plans to submit to the EMA in the middle of 2015 under the Centralised Procedure.

"The submission of our marketing application in Europe will be an important milestone in our global strategy to get migalastat approved for Fabry patients with amenable mutations as quickly as possible," said John F. Crowley, chairman and chief executive officer of Amicus Therapeutics. "We believe significant unmet need still exists in the treatment of Fabry disease and that migalastat could become a very important, differentiated oral therapy. We also look forward to meeting with the FDA in the first quarter of 2015 to discuss the US regulatory path."

As previously reported, migalastat successfully met both co-primary endpoints of comparability to enzyme replacement therapy (ERT) on both key measures of kidney function in a phase 3 global registration study (Study 012) that compared migalastat to standard-of-care ERTs in Fabry patients with amenable mutations. Study 012 was designed based on feedback from the EMA. The Company has also previously announced positive 12- and 24-month results from a phase 3 global registration study (Study 011) that demonstrated the benefit of migalastat compared to placebo in Fabry patients with amenable mutations.

Study 012 was a phase 3, open-label study that compared oral migalastat to standard-of-care ERTs for Fabry disease (Fabrazyme and Replagal). 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, after which they were eligible to receive migalastat in a 12-month extension phase.

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 cardiac function assessed by echocardiography, as well as a composite of Fabry-associated clinical events (i.e. renal, cardiac, or cerebrovascular).

Amicus Therapeutics is a biopharmaceutical company at the forefront of therapies for rare and orphan diseases. The Company is developing novel, first-in-class treatments for a broad range of human genetic diseases, with a focus on delivering new benefits to individuals with lysosomal storage diseases. Amicus' lead programs include the small molecule pharmacological chaperones migalastat as a monotherapy and in combination with enzyme replacement therapy (ERT) for Fabry disease; and AT2220 (duvoglustat) in combination with ERT for Pompe disease.