Advice - reimburse olipudase alfa (Xenpozyme®) for the treatment of Niemann-Pick disease
The National Health Care Institute has advised the Minister of Health, Welfare and Sport (VWS) to reimburse olipudase alfa (Xenpozyme®) from the basic health care package after price negotiations. This medicinal product can be used for certain patients with Niemann-Pick disease. This is a hereditary metabolic disease. The reason for this advice was the placing of the medicinal product in the so-called ‘lock procedure for expensive medicinal products’.
Olipudase alfa is intended for certain people with Niemann-Pick disease
Olipudase alfa is the active substance. The brand name is Xenpozyme®. The medicinal product is administered via an IV into the blood.
The product can be used in people with Niemann-Pick disease type A/B and type B. This is a very rare hereditary metabolic disorder in which a person produces less of a certain enzyme and the enzyme thus works less well. This prevents the break-down of certain lipids (sphingolipids). This leads to damage in organs, such as the spleen, liver and lungs, and at worst the nervous system. Patients may experience anaemia, bleeding, liver failure, high cholesterol, lung infections, tightness of the chest, exhaustion and a higher risk of death at an early age. Patients with type B never have neurological symptoms and have a normal mental development. This is different from patients with type A/B; they do suffer from neurological symptoms, such as developmental delay.
Advice from the National Health Care Institute on the reimbursement of olipudase alfa
The National Health Care Institute advises the Minister of Health, Welfare and Sport to reimburse olipudase alfa (Xenpozyme®) 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 Scientific Advisory Board (WAR) and the Insured Package Advisory Committee (ACP) advise the National Health Care Institute about the assessment. The Minister makes the final decision whether or not to reimburse the medication from the basic health care package.
Lock procedure for expensive medicinal products
The Minister has placed olipudase 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’.