Spinraza (nusinersen) is indicated for the treatment of pediatric and adult patients with spinal muscular atrophy (SMA). It is injected into the spinal fluid every few months and costs roughly €82,500 per 5ml vial for one treatment. In clinical trials, treatment with this medicine has been shown to slow down disease progression and improve motor function.
As SMA's symptoms worsen over time, it is important to begin treatment as soon as possible in order to preserve motor function. The longer it takes for this medicine to become approved or available, the worse a patient's condition will become. This therapy is administered for as long as the patient responds to treatment. Given that the most common types of SMA affect infants and is a lifelong condition, it is difficult for many families to afford this therapy without financial assistance.
Types of spinal muscular atrophy
Spinal muscular atrophy (SMA) is a genetic disease that causes weakness and muscle wasting due to the loss of nerve cells, called motor neurons, that control movement. Different types of SMA are classified based on changes in a particular gene, the patient's age at the onset of the disease and the severity of muscle weakness.
SMA type 1: onset before 6 months, most common type
SMA type 2: onset between 6 and 18 months
SMA type 3: generally milder, onset is usually late but can be as early as at 12 months
-Type 3a: onset before age of 3
-Type 3b: onset after 3 years and usually slower progression
SMA type 4: adult onset, usually after 35 years old, generally mildest and least common
To learn more about this disease how the treatment works, please read our earlier article entitled Spinraza, the new medicine for spinal muscular atrophy.
Spinraza (nusinersen) is manufactured by the pharmaceutical company, Biogen. In most countries where approved, this medicine has been approved for the treatment of patients with types 1-3. Although some countries have approved Spinraza (nusinersen), not all of them have made it available. You can learn more about Spinraza (nusinersen) here.
Usually, countries must first approve the medicine before the ministry of health tackles the issue of reimbursement. To date, over 50 countries have already agreed to reimburse or are negotiating reimbursement of this medicine. However, the criteria for reimbursement varies from country to country with some offering full reimbursement for all SMA types while others refund only patients with type I, for example.
Is Spinraza (nusinersen) reimbursed in my country?
If you would like to know more about availability or reimbursement in your country, please contact us - our support team will be happy to provide you with more information.
In the instances where Spinraza (nusinersen) is not available through the local Ministry of Health (even if reimbursement is offered), it can still be obtained through the Named Patient personal import basis which allows patients in urgent need to access these types of medicines.
To illustrate the differences in the way that countries give compensation, below you will find three examples of countries where Spinraza (nusinersen) is reimbursed and available, reimbursed but not available, and where it is not reimbursed or available. This is followed by a comprehensive list of countries and their reimbursement status.
3 Countries where Spinraza (nusinersen) is reimbursed and available
Italy: reimbursed access; for patients affected by SMA types I, II and III; with no age limits
Norway: reimbursed access; for patients affected by SMA types I, II and IIIa; 0 to 18 years of age
Netherlands: reimbursed access; for specified groups of patients with SMA types I, II and IIIa
-Type I, infantile-onset: 0-6 months, diagnosed not longer than 26 weeks before initiation of treatment
-Type II and IIIa, later onset: 6 to 20 months, diagnosed not longer than 94 months before initiation of treatment
-Presymptomatic infants diagnosed on the basis of genetic tests.
3 countries where Spinraza (nusinersen) is reimbursed but not available
Romania: reimbursed access in line with the label (October 2018); for patients affected by 5q spinal muscular atrophy (SMA)
-Currently not available in Romanian market, patients seeking medicine in Italy
Croatia: reimbursed access; for patients affected by SMA types I, II and III; less than 18 years of age; who are not on ventilation
-Not available through the Croatian Health Insurance Fund (HZZO) that manages their universal health care system.
Poland: reimbursed access; for patients affected by all SMA types; with no age limits
-Effective January 1, 2019
-Hospitals in process of arranging paperwork before medicine deliveries can be made.
3 countries where Spinraza (nusinersen) is not reimbursed nor available
Brazil: reimbursement proposal refused
-According to the Secretária de Vigilância em Saúde, the Brazilian Ministry of Health, Spinraza (nusinersen) is approved but not available through due to a lack of clear positive results
-Patients currently pay full price for medicines or opt to sue Brazil's Ministry of Health in order to split the cost.
United Kingdom: not reimbursed by the NHS, except in Scotland for type I SMA
-The Scottish Medicines Consortium is considering wider use under its recently established “ultra-orphan treatment” category.
Turkey: reimbursement negotiations underway
-Early access programme available for patients with SMA type I; who are not on ventilation.
The following countries offer reimbursement access in line with the label ‐ 5q spinal muscular atrophy (SMA), or more.
These countries have reimbursement restrictions based on age, SMA type or other criteria.
No reimbursement - negotiations or preparations underway
No refunds are currently available in these countries, in some cases reimbursement negotiations are underway.
United Arab Emirates
No refunds are currently available in these countries.
...all other countries
*Table based on information from SMA Support UK
**All information in this article is current as of the time of writing, to the best of our ability. Please contact us for the latest updates.
- Spinraza (nusinersen). Official website.
- SMA Types. SMA News Today. Accessed December 2018.
- FDA Approves Biogen’s SPINRAZA™ (nusinersen), The First Treatment for Spinal Muscular Atrophy. Biogen, Dec 2016. Accessed December 2018.
- Biogen Perspective on Innovative Pricing & Contracting [PDF]. SMA Support UK. Accessed December 2018.
- Biogen SMA Community Update UK October 2018 [PDF]. SMA Support UK.Accessed December 2018.