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  • Amicus Therapeutics announces European Commission ...

Amicus Therapeutics announces European Commission approval of Galafold for adolescents with Fabry Disease.

Read time: 1 mins
Published:12th Aug 2021
Amicus Therapeutics has announced the European Commission has approved Galafold (migalastat) for use in adolescents aged 12 to <16 years weighing greater than 45 kg with a confirmed diagnosis of fabry disease and who have an amenable mutation.>

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).

Condition: Fabry Disease
Type: drug

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