Amicus Therapeutics has held meetings with the EMA and FDA to discuss approval pathways for migalastat as monotherapy for Fabry disease. The assigned European rapporteurs have acknowledged that Amicus Therapeutics followed the EMA's recommendations for clinical development and have generated data of significant quality and quantity in the phase III trials FACETS (ClinicalTrials.gov Identifier NCT00925301) and ATTRACT (ClinicalTrials.gov Identifier NCT01218659).
The rapporteurs also suggested that the company should request accelerated assessment, given the need for treatment options in Fabry disease. Amicus Therapeutics now intends to file an MAA during the second quarter of 2015. Following a meeting with the FDA, the company intends to schedule a pre-NDA meeting and to file an NDA under the Accelerated Approval (Subpart H) regulations in the second half of 2015. This means that there will be a requirement for a phase IV study.
The FDA has indicated that it will consider surrogate endpoints of Fabry disease in its approval decision. These include reduction of interstitial capillary globotriaosylceramide (GL-3), reduction in cardiac mass and stabilization of glomerular filtration rate, which were measured in the phase III trials (Amicus Therapeutics News Release).