GVS advice reassessment migalastat (Galafold®) for the treatment of Fabry disease

Condition for which reimbursement is requested

Migalastat is used for the long-term treatment of patients aged 12 years and older with a confirmed diagnosis of Fabry disease (alpha-galactosidase A deficiency) and an amenable mutation.

In Fabry disease, the enzymes that transport waste from cells are ineffective or less effective. As a result, waste accumulates in the wall of the blood vessels and in the cells of various organs, such as the heart and kidneys. This interferes with the functioning of organs and can lead to life-threatening problems later in life. Because Fabry is a congenital disease, symptoms already develop in childhood. However, early symptoms, such as nausea and pain attacks, are not specific to this disease. That makes early identification and treatment more difficult. At a later age, kidneys, heart and brain often do not work well. In men with Fabry disease, life expectancy is 20 years lower than normal. In women, this is 15 years.

Previous assessment of the National Health Care Institute

In April 2017, the National Health Care Institute ruled that migalastat did not meet the 'established medical science and medical practice', compared with usual enzyme replacement therapy (ERT), agalsidase alfa (Replagal®) and agalsidase beta (Fabrazyme®). This is the legal criterion on the basis of which health care may be reimbursed from the basic health care package. Based on new study data, the marketing authorisation holder has requested a reassessment of migalastat. 

Read the previous assessment of migalastat of April 2017.

Recommendations from the National Health Care Institute

The National Health Care Institute advises the Minister of Health, Welfare and Sport (VWS) not to include migalastat in the basic health care package for the treatment of patients aged 12 years and older with Fabry disease who have an amenable mutation and an estimated glomerular filtration rate (eGFR) >30 ml/min/1.73 m2.

Medicine Reimbursement System (GVS)

Migalastat is an outpatient medicinal product. Outpatient medicinal products are medicinal products for home use, available at the pharmacy on prescription from a physician. They will only be reimbursed from the basic health care package if they are listed in the GVS. The amount of the reimbursement depends on the list the product is on. Interchangeable medicinal products are clustered in List 1A. These may be subject to a reimbursement limit. Unique medicinal products are listed in List 1B. There is no reimbursement limit for these products. Additional conditions may apply for reimbursement. In that case, a medicinal product is also listed on List 2.

The Scientific Advisory Board (WAR) advises the National Health Care Institute about the assessment. Based on the assessment, the National Health Care Institute sends an advisory report to the Minister of Health, Welfare and Sport. The Minister makes the final decision whether or not to reimburse the medication from the basic health care package.

This report is a summary of recommendations by the National Health Care Institute. The original text is in Dutch.