Galafold reimbursed as first-line drug for Fabry disease

May be reimbursed as a first-line treatment without receiving prior enzyme replacement therapy (ERT)

Insurance reimbursement age expanded from 16 or older to 12 years or older

Reimbursement for Handok's Fabry disease treatment, Galafold (migalastat), will be expanded to cover its use as a first-line treatment starting on the first of this month.

 

Previously, Galafold was covered only for patients aged 16 and older who had been administered enzyme replacement therapy (ERT) intravenously for at least 12 months.

 

With this expansion of reimbursement, Galafold can now be prescribed as a first-line treatment without 12 months of enzyme replacement therapy.

 

In addition, as stated in the approval, patients aged 12 years and older (weighing 45 kg or more) will be eligible for insurance reimbursement.

 

Galafold is the world's first oral treatment for Fabry disease, developed by Amicus and supplied by Handok in South Korea.

 

It is currently used in 45 countries, including the United States, the United Kingdom, Australia, Japan, and Europe, and was approved in Korea in 2017 and became listed for reimbursement in 2019.

 

Unlike enzyme replacement therapy, which requires patients to visit the hospital once every two weeks for intravenous injections lasting several hours, Galafold is an oral treatment that patients can take themselves once every two days.

 

This improves the convenience of medication administration and quality of life for Fabry disease patients.

 

Galafold demonstrated a sustained and long-term safety profile in maintaining kidney function and reducing cardiac mass index when switching from enzyme replacement therapy to Galafold in Fabry disease patients with a compliant genotype, as confirmed by a 30-month long-term clinical trial.

 

Additionally, in the switching group, cardiac adverse reactions occurred in 20% of patients receiving enzyme replacement therapy, but the rate decreased to 7% after switching to Galafold.

 

Galafold demonstrated efficacy and safety not only in patients switching from enzyme replacement therapy but also in treatment-naive patients.

 

In the Phase III FACET study, a 24-month clinical trial of Galafold in treatment-naive or enzyme replacement therapy-naive patients with Fabry disease harboring a compliant mutation, cardiac mass index decreased significantly from baseline, with a greater reduction in patients with left ventricular hypertrophy.

 

(Overall: -7.7 ± 3.7 g/m², patients with left ventricular hypertrophy: -18.6 ± 8.3 g/m²) Additionally, in the long-term real-world patient registry study followMe , Galafold maintained stable renal function over an average treatment duration of 3.9 years, and that Fabry disease-related clinical events occurred in less than 80% of patients, confirming renal preservation and multi-organ benefits.

 

Recently, the results of the ASPIRE study in pediatric patients were published in the May issue of the international academic journal ‘Molecular Genetics and Metabolism.’ The ASPIRE study showed that pediatric patients treated with Galafold maintained stable renal and cardiac markers, plasma lyso-Gb3 levels, and improvements in pain, gastrointestinal symptoms, and quality of life.

 

Additionally, no new adverse reactions or unexpected safety issues were observed in the safety evaluation.