Biotechnology company Amicus Therapeutics has been granted complete approval by the European Commission (EC) for its oral, small molecule, pharmacological chaperone Galafold (migalastat).
Galafold (migalastat) serves as a first line therapy for long-term treatment of adults, as well as adolescents of 16 years of age and above affected with Fabry disease (alpha-galactosidase A deficiency) and who have an amenable mutation.
Amicus Therapeutics chairman and chief executive officer John F. Crowley said: "This EU approval for Galafold is a significant advancement in the field of precision genetic medicine and a tremendous milestone for the Fabry community."
The company recently commenced its supply of Galafold in the German market.
Galafold (migalastat) is the first oral treatment, as well as the first precision medicine available to treat the Fabry disease.
The approval from EC was based on clinical data from two Phase III pivotal studies.
University College London UK senior lecturer Derralynn Hughes said: "As principal investigator in both Galafold pivotal studies, I have experience treating both naïve and treatment-experienced Fabry patients with Galafold.
"I am pleased that the European Commission has approved this new treatment option and I believe it has the potential to address unmet needs among Fabry patients who have amenable mutations."
A rare genetic and life-threatening disease, Fabry is caused by the accumulation of disease substrate (globotriaosylceramide, GL-3) in the lysosome due to a dysfunctional or deficient enzyme.
Galafold helps stabilise the body’s own dysfunctional enzyme, so that it can clear the accumulation of disease substrate in patients who have amenable mutations, which is responsive to therapy with Galafold based on predefined criteria.
Image: Alpha galactosidase, the deficient protein Fabry disease. Photo: courtesy of ProteinBoxBot / English Wikipedia.