The European Commission has extended the use of Galafold (migalastat) to children from 12 years of age and weighing at least 45 kilograms (99 pounds), with confirmed Fabry disease and a genetic mutation known to be sensitive to treatment .

Galafold, developed by Amicus Therapeutics, is approved in over 40 countries. The treatment was first approved in the European Union for these Fabry patients, aged 16 and over, in 2016. In the United States, the treatment is used only in adults.

“This Galafold approval is a transformative moment for the Fabry community in the EU [European Union]because it offers patients as young as 12 years old with a susceptible mutation a new treatment option for the first time in over 15 years, ”said Bradley Campbell, President and COO of Amicus Therapeutics , in a press release.

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“This expanded endorsement is an important step forward for the Fabry community as we work to increase awareness of this rare disease among young people. It ensures that patients, both pediatric and adult, now have a convenient treatment option for oral disease modification, ”said Uma Ramaswami, MD, Lysosomal Storage Disease Unit, Royal Free London NHS Foundation Trust.

Fabry is caused by mutations in the GLA gene, which leads to a defective alpha-galactosidase A (Gal A) enzyme. This enzyme works to break down a type of fat called globotriaosylceramide (Gb3) into building blocks that cells can use.

By restoring the activity of the defective Gal A enzyme, Galafold is designed to remove the buildup of Gb3 which is toxic to cells and tissues. It is a chaperone therapy that binds to mutant “acceptable” forms of alpha-GAL A, making their structure more stable and partially restoring enzyme activity. Available as 123 mg capsules, Galafold has been approved for use in the EU for over 1000 GLA mutations.

The expansion of the European label was supported by one-month interim data from the recently completed study AT1001-020 (NCT03500094), a phase 3b open-label trial that evaluated Galafold in 22 adolescents, ages 12 to 17 years old, with sensitive mutations.

The study was divided into two parts. First, the participants were treated with one 123 mg capsule of Galafold every other day for four weeks (one month), after which their safety and effectiveness were evaluated. Then, without interruption of treatment, patients entered part 2 and received Galafold for an additional 11 months, for a total of one year of treatment. This was followed by a 30-day no-treatment safety follow-up period.

Its objective was to assess the safety, pharmacokinetics, pharmacodynamics and efficacy of the therapy. It should be noted that pharmacokinetics refers to the movement of a drug in, through, and out of the body, while pharmacodynamics is defined as the effects of a compound on the body.

“We are grateful to this wonderful and passionate community, especially the patients, physicians and families who made this research possible through their participation in clinical trials, as well as the regulators and our dedicated Amicus team,” said Campbell. “This achievement highlights our company’s mission, which is focused on transforming the lives of people living with rare diseases. “

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