Amicus To Submit EMA For Migalastat Monotherapy In Fabry Disease

(RTTNews) - Amicus Therapeutics (FOLD) Thursday said it has conducted a successful pre-submission meeting with the European Medicines Agency or EMA to discuss the registration of migalastat HCl 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 or MAA, which it plans to submit to the EMA in the middle of 2015 under the Centralized Procedure.

The firm also looks forward to meeting with the FDA in the first quarter of 2015 to discuss the U.S. regulatory path.

Migalastat had successfully met both co-primary endpoints of comparability to enzyme replacement therapy or ERT on both key measures of kidney function in a Phase 3 global registration study that compared migalastat to standard-of-care ERTs in Fabry patients with amenable mutations.