New Medicines

GalafoldFabry's disease in patients aged 12–15 years old


Licence extension / variation
Amicus Therapeutics
Amicus Therapeutics

Development and Regulatory status

Phase III Clinical Trials
May 2022
May 22MHRA approves a licence change for Galafold to extend use to adolescents (previously only adults). The revised indication is long-term treatment of adults and adolescents, aged ≥12 years and weighing ≥45kg, with a confirmed diagnosis of Fabry disease (α-galactosidase A deficiency) and who have an amenable mutation [7].
Aug 21Approved in EU for adolescents aged 12 to <16 years weighing ≥ 45 kg with Fabry disease and with an amenable mutation [6].
Jun 21Extension to existing indication recommended for EU approval by CHMP – the new indication is “Galafold is indicated for long-term treatment of adults and adolescents aged 12 years and older with a confirmed diagnosis of Fabry disease (α-galactosidase A deficiency) and who have an amenable mutation [6].
Jan 19Has orphan drug status in EU & US [3].


Alpha-2 galactosidase stimulant
Anderson-Fabry disease is an X-linked recessive inborn error of glycosphingolipid metabolism caused by deficiency of alpha-galactosidase A. It is estimated to occur in 1 in 55,000 males in the classical form but the atypical variant may be more common [1].
Fabry's disease in patients aged 12–15 years old

Trial or other data

Dec 19NCT03500094 estimated primary completion now March 2021; recruitment also includes UK and Spain [5].
Oct 19Estimated start for PIII study NCT04049760, a long-term follow-up study to NCT03500094; patients will transfer to the extension study and be followed for up to five years. It is taking place in the US and Spain. Primary outcomes include safety and clinical measures, and estimated primary outcome date is June 2022 [4].
Oct 18PIII study (NCT03500094) to evaluate the safety, pharmacokinetics, pharmacodynamics, and efficacy of migalastat treatment in pediatric subjects 12 to <18 years of age with Fabry disease and amenable GLA variants starts. Subjects must be either naïve to enzyme replacement therapy (ERT) or have stopped ERT at least 14 days at the time of screening. Stage 1 will be a treatment period of approximately 1 month (4 weeks); Stage 2 will be a treatment period of 11 months and a 30-day (untreated) safety follow-up period. There will be no break in treatment between Stages 1 and 2. Prior to Stage 1, there will be a screening period lasting at least 14 days and up to 30 days (or more, if GLA genotyping is required). Stage 1 and 2 together will consist of a 12-month treatment period, and a 30-day safety follow-up period, for a total of approximately 13 months. Subjects may have the option to enroll in a long-term extension study conducted under a separate protocol. Subjects will be randomly assigned 1:1:1 to 1 of 3 PK sampling groups using interactive response technology (IRT). Four blood samples for determination of migalastat concentrations in plasma will be collected on any one 24 hour period between Day 15 and Day 30 of study drug administration and again at Months 6 and 12. 20 patients will be recruited in the US, & collection of primary outcome data is due to complete Sep 20 [2].

Evidence based evaluations