Advice - reimburse pegunigalsidase alfa (Elfabrio®) for the treatment of Fabry disease

The National Health Care Institute has advised the Minister for Medical Care to reimburse pegunigalsidase alfa (Elfabrio®) from the basic health care package, after price negotiations. This medicinal product can be used for certain patients with Fabry disease. The reason for this advice was the placing of the medicinal product in the so-called ‘lock procedure for expensive medicinal products’. 

Pegunigalsidase alfa is intended for certain people with Fabry disease

Pegunigalsidase alfa is the active substance. The brand name is Elfabrio®. The medicinal product is administered by infusion. 

This medicinal product can be used for patients with Fabry disease. This disease is also called alpha-galactosidase A deficiency. In Fabry disease, the enzymes that transport waste from cells are inactive or less active. As a result, waste accumulates in the wall of the blood vessels and in the cells of various organs, such as heart and kidneys. This interferes with the functioning of organs and can lead to life-threatening problems later in life. Because Fabry disease 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.

Advice from the National Health Care Institute on the reimbursement of pegunigalsidase alpha

The National Health Care Institute advises the Minister for Medical Care to reimburse pegunigalsidase alfa (Elfabrio®) from the basic health care package after price negotiations. 

More information or questions?

If you have any questions about this advice, please send your question to the National Health Care Institute via warcg@zinl.nl. If you have questions about the reimbursement of a medicinal product, the personal expenses or whether you should pay a contribution, please ask your health insurance provider.

How did the advice come about?

The National Health Care Institute advises the Minister about the assessment. The final decision as to whether or not reimbursement from the basic health care package will take place lies with the Minister.

Lock procedure for expensive medicinal products

The Minister has placed pegunigalsidase alfa for this indication in the lock procedure for expensive medicinal products. A medicinal product in this lock procedure will not be eligible for reimbursement from the basic health care package until:

  • there is a positive package advice from the National Health Care Institute;
  • there are arrangements and safeguards for appropriateness in place;
  • price reductions have been successfully negotiated with the manufacturer.

For more information, see the page ‘Lock procedure for expensive medicinal products’.

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