Amicus Therapeutics submits MAA to EMA for Galafold (migalastat HCl) to treat genotype of Fabry disease.

Amicus Therapeutics has submitted a marketing authorization application (MAA) to request full approval of the oral small molecule pharmacological chaperone Galafold (migalastat HCl) for Fabry patients who have amenable genetic mutations. The brand name Galafold has been approved by both the European Medicines Agency (EMA) as well as the FDA. The drug was previously known as Amigal. If approved, Amicus estimates that 30% to 50% of Fabry patients may benefit from Galafold on the basis of their genotype. Based on third party research, the Company also projects a 10% annual growth rate in diagnosis that is expected to continue.

Approximately 90 individuals around the world are currently being treated with Galafold as their only therapy for Fabry disease in ongoing long-term extension studies. Amicus previously reported positive Phase III data for Galafold in both treatment na�ve (Study 011, or FACETS) and enzyme replacement therapy (ERT) switch patients (Study 012, or ATTRACT). Results from these studies have shown that treatment with Galafold has resulted in reductions in disease substrate, stability of kidney function, reduction in cardiac mass, and a positive impact on patient-reported outcomes in patients with amenable mutations. Today approximately 5,000 to 10,000 individuals are diagnosed with Fabry disease, however, an estimated 40% to 50% of diagnosed patients are not currently on treatment. If approved, Amicus estimates that 30% to 50% of Fabry patients may benefit from Galafold on the basis of their genotype.

Comment: Amicus has developed a novel approach to address human genetic diseases resulting from misfolded proteins- pharmacological chaperones- to restore protein function and eliminate aggregation of misfolded proteins. Formerly allied with GSK which had provided funding, Amicus is now proceeding alone with the applications to EMA and the FDA but with a commitment to provide milestone payments to GSK which has taken a $3 million investment in Amicus as part of its withdrawal. Amicus also plans to conduct a pre-NDA meeting with the US FDA and to submit a New Drug Application (NDA) for Galafold in the United States under Subpart H1 in the second half of 2015