Amicus Therapeutics announces European Commission approval of Galafold for adolescents with Fabry Disease.
Galafold is already approved in multiple geographies around the world, including the u.s., eu, and japan, for adults who have an amenable variant, or mutation.></16>
The extension of the indication was supported by 1-month interim safety and pharmacokinetics data from Study AT1001-020 which was a 2-stage, open-label, multicenter study to evaluate the safety, pharmacokinetics, pharmacodynamics, and efficacy of migalastat treatment in pediatric subjects aged 12 to less than 18 years and weighing greater than 45 kg with Fabry disease and with amenable mutations to the gene encoding ?-galactosidase A (GLA).
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