Spinraza

nusinersen

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This is a summary of the European public assessment report (EPAR) for Spinraza. It explains how the Agency assessed the medicine to recommend its authorisation in the EU and its conditions of use. It is not intended to provide practical advice on how to use Spinraza.

For practical information about using Spinraza, patients should read the package leaflet or contact their doctor or pharmacist.

What is Spinraza and what is it used for?

Spinraza is a medicine used to treat 5q spinal muscular atrophy (SMA), a genetic disease that causes weakness and wasting of the muscles including the lung muscles. The disease is linked to a defect on chromosome 5q and symptoms usually start shortly after birth.

Because the number of patients with SMA is low, the disease is considered ‘rare’, and Spinraza was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 2 April 2012.

Spinraza contains the active substance nusinersen.

How is Spinraza used?

Spinraza can only be obtained with a prescription and treatment should be started by a doctor with experience in the treatment of SMA.

The medicine is available as a solution for injection in 12 mg vials. It is given by intrathecal injection (into the lower back, directly into the spine) by a doctor or nurse experienced in carrying out this procedure. The patient may need to be sedated (given a medicine to calm them) before they are given Spinraza.

The recommended dose is 12 mg (one vial), given as soon as possible after the patient has been diagnosed with SMA. The first dose should be followed by 3 more doses after 2, 4, and 9 weeks and then one dose every 4 months thereafter. Treatment should be continued for as long as the patient benefits from it. For further information, see the package leaflet.

How does Spinraza work?

Patients with SMA lack a protein called ‘survival motor neuron’ (SMN) protein, which is essential for motor neurons (nerve cells from the spinal cord that control muscle movements) to survive and function normally. The SMN protein is made from two genes, SMN1 and SMN2. Patients with SMA lack the SMN1 gene but have the SMN2 gene, which mostly produces a short SMN protein that does not work as well as a full-length protein.

Spinraza is a synthetic anti-sense oligonucleotide (a type of genetic material) that enables the SMN2 gene to produce full length protein, which is able to work normally. This replaces the missing protein, thereby relieving the symptoms of the disease.

What benefits of Spinraza have been shown in studies?

One main study, involving 121 babies (of an average age of 7 months) with SMA, showed that Spinraza is effective in improving movement when compared to placebo (sham injection).

After one year of treatment, 51% of babies receiving Spinraza (37 out of 73) showed progress in developing head control, rolling, sitting, crawling, standing and walking, whereas no similar progress was seen in any of the babies who received placebo. In addition, most babies treated with Spinraza survived for longer and needed breathing support later than those given placebo.

Another study is ongoing to assess Spinraza’s effectiveness in children whose SMA was less severe and was diagnosed at a later stage (average age of 3 years). Interim analysis has shown results consistent with those in babies where the disease started earlier.

What are the risks associated with Spinraza?

The most common side effects with Spinraza (which may affect more than 1 in 10 people) are headache and back pain; however in babies these side effects could not be assessed, as they could not communicate them. These side effects are thought to be caused by the injections into the spine used to give the medicine.

For the full list of all side effects and restrictions with Spinraza, see the package leaflet.

Why is Spinraza approved?

In its assessment, the Committee for Medicinal Products for Human Use (CHMP) recognised the serious nature of the disease and the urgent need for effective treatments.

Spinraza has been shown to lead to clinically meaningful improvements in young children with varying degrees of disease severity. Although the medicine was not tested in patients with the most severe and the mildest forms of SMA, it is expected to provide similar benefits to these patients.

Side effects were considered manageable, with most side effects related to the way the medicine is given.

The CHMP therefore decided that Spinraza’s benefits are greater than its risks and recommended that it be approved for use in the EU.

What measures are being taken to ensure the safe and effective use of Spinraza?

The company that markets Spinraza will complete ongoing studies of the long-term safety and effectiveness of the medicine in patients who are showing symptoms of SMA and patients not yet showing symptoms.

Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Spinraza have also been included in the summary of product characteristics and the package leaflet.

Other information about Spinraza

The European Commission granted a marketing authorisation valid throughout the European Union for Spinraza on 30 May 2017.

For more information about treatment with Spinraza, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.

Name Language First published Last updated
Spinraza : EPAR - Summary for the public BG = bălgarski 2017-06-21  
Spinraza : EPAR - Summary for the public ES = español 2017-06-21  
Spinraza : EPAR - Summary for the public CS = čeština 2017-06-21  
Spinraza : EPAR - Summary for the public DA = dansk 2017-06-21  
Spinraza : EPAR - Summary for the public DE = Deutsch 2017-06-21  
Spinraza : EPAR - Summary for the public ET = eesti keel 2017-06-21  
Spinraza : EPAR - Summary for the public EL = elliniká 2017-06-21  
Spinraza : EPAR - Summary for the public EN = English 2017-06-21  
Spinraza : EPAR - Summary for the public FR = français 2017-06-21  
Spinraza : EPAR - Summary for the public IT = italiano 2017-06-21  
Spinraza : EPAR - Summary for the public LV = latviešu valoda 2017-06-21  
Spinraza : EPAR - Summary for the public LT = lietuvių kalba 2017-06-21  
Spinraza : EPAR - Summary for the public HU = magyar 2017-06-21  
Spinraza : EPAR - Summary for the public MT = Malti 2017-06-21  
Spinraza : EPAR - Summary for the public NL = Nederlands 2017-06-21  
Spinraza : EPAR - Summary for the public PL = polski 2017-06-21  
Spinraza : EPAR - Summary for the public PT = português 2017-06-21  
Spinraza : EPAR - Summary for the public RO = română 2017-06-21  
Spinraza : EPAR - Summary for the public SK = slovenčina 2017-06-21  
Spinraza : EPAR - Summary for the public SL = slovenščina 2017-06-21  
Spinraza : EPAR - Summary for the public FI = suomi 2017-06-21  
Spinraza : EPAR - Summary for the public SV = svenska 2017-06-21  
Spinraza : EPAR - Summary for the public HR = Hrvatski 2017-06-21  

This EPAR was last updated on 21/06/2017 .

Authorisation details

Product details

Product details for Spinraza
NameSpinraza
Agency product numberEMEA/H/C/004312
Active substance

nusinersen sodium

International non-proprietary name (INN) or common name

nusinersen

Therapeutic area Muscular Atrophy, Spinal
Anatomical therapeutic chemical (ATC) code N07
Additional monitoring

This medicine is under additional monitoring. This means that it is being monitored even more intensively than other medicines. For more information, see medicines under additional monitoring.

Treatment of rare diseases

This medicine has an "orphan designation" which means that it is used to treat life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in the European Union, or are medicines which, for economic reasons, would be unlikely to be developed without incentives.

Publication details

Publication details for Spinraza
Marketing-authorisation holder

Biogen Idec Ltd

Revision0
Date of issue of marketing authorisation valid throughout the European Union30/05/2017

Contact address:

Biogen Idec Ltd
Innovation House  
70 Norden Road 
Berkshire 
Maidenhead 
SL6 4AY 
United Kigndom

Product information

Product information

30/05/2017  Spinraza -EMEA/H/C/004312 --

Name Language First published Last updated
Spinraza : EPAR - Product Information HR = Hrvatski 2017-06-21  
Spinraza : EPAR - Product Information HR = Hrvatski 2017-06-21  
Spinraza : EPAR - Product Information HR = Hrvatski 2017-06-21  
Spinraza : EPAR - Product Information HR = Hrvatski 2017-06-21  
Spinraza : EPAR - Product Information HR = Hrvatski 2017-06-21  
Spinraza : EPAR - Product Information HR = Hrvatski 2017-06-21  
Spinraza : EPAR - Product Information HR = Hrvatski 2017-06-21  
Spinraza : EPAR - Product Information HR = Hrvatski 2017-06-21  
Spinraza : EPAR - Product Information HR = Hrvatski 2017-06-21  
Spinraza : EPAR - Product Information HR = Hrvatski 2017-06-21  
Spinraza : EPAR - Product Information HR = Hrvatski 2017-06-21  
Spinraza : EPAR - Product Information HR = Hrvatski 2017-06-21  
Spinraza : EPAR - Product Information HR = Hrvatski 2017-06-21  
Spinraza : EPAR - Product Information HR = Hrvatski 2017-06-21  
Spinraza : EPAR - Product Information HR = Hrvatski 2017-06-21  
Spinraza : EPAR - Product Information HR = Hrvatski 2017-06-21  
Spinraza : EPAR - Product Information HR = Hrvatski 2017-06-21  
Spinraza : EPAR - Product Information HR = Hrvatski 2017-06-21  
Spinraza : EPAR - Product Information HR = Hrvatski 2017-06-21  
Spinraza : EPAR - Product Information HR = Hrvatski 2017-06-21  
Spinraza : EPAR - Product Information HR = Hrvatski 2017-06-21  
Spinraza : EPAR - Product Information HR = Hrvatski 2017-06-21  
Spinraza : EPAR - Product Information HR = Hrvatski 2017-06-21  
Spinraza : EPAR - Product Information HR = Hrvatski 2017-06-21  
Spinraza : EPAR - Product Information HR = Hrvatski 2017-06-21  

Contents

  • Annex I - Summary of product characteristics
  • Annex IIA - Manufacturing-authorisation holder responsible for batch release
  • Annex IIB - Conditions of the marketing authorisation
  • Annex IIIA - Labelling
  • Annex IIIB - Package leaflet

Please note that the size of the above document can exceed 50 pages.

You are therefore advised to be selective about which sections or pages you wish to print.


Name Language First published Last updated
Spinraza : EPAR - All Authorised presentations HR = Hrvatski 2017-06-21  
Spinraza : EPAR - All Authorised presentations HR = Hrvatski 2017-06-21  
Spinraza : EPAR - All Authorised presentations HR = Hrvatski 2017-06-21  
Spinraza : EPAR - All Authorised presentations HR = Hrvatski 2017-06-21  
Spinraza : EPAR - All Authorised presentations HR = Hrvatski 2017-06-21  
Spinraza : EPAR - All Authorised presentations HR = Hrvatski 2017-06-21  
Spinraza : EPAR - All Authorised presentations HR = Hrvatski 2017-06-21  
Spinraza : EPAR - All Authorised presentations HR = Hrvatski 2017-06-21  
Spinraza : EPAR - All Authorised presentations HR = Hrvatski 2017-06-21  
Spinraza : EPAR - All Authorised presentations HR = Hrvatski 2017-06-21  
Spinraza : EPAR - All Authorised presentations HR = Hrvatski 2017-06-21  
Spinraza : EPAR - All Authorised presentations HR = Hrvatski 2017-06-21  
Spinraza : EPAR - All Authorised presentations HR = Hrvatski 2017-06-21  
Spinraza : EPAR - All Authorised presentations HR = Hrvatski 2017-06-21  
Spinraza : EPAR - All Authorised presentations HR = Hrvatski 2017-06-21  
Spinraza : EPAR - All Authorised presentations HR = Hrvatski 2017-06-21  
Spinraza : EPAR - All Authorised presentations HR = Hrvatski 2017-06-21  
Spinraza : EPAR - All Authorised presentations HR = Hrvatski 2017-06-21  
Spinraza : EPAR - All Authorised presentations HR = Hrvatski 2017-06-21  
Spinraza : EPAR - All Authorised presentations HR = Hrvatski 2017-06-21  
Spinraza : EPAR - All Authorised presentations HR = Hrvatski 2017-06-21  
Spinraza : EPAR - All Authorised presentations HR = Hrvatski 2017-06-21  
Spinraza : EPAR - All Authorised presentations HR = Hrvatski 2017-06-21  
Spinraza : EPAR - All Authorised presentations HR = Hrvatski 2017-06-21  
Spinraza : EPAR - All Authorised presentations HR = Hrvatski 2017-06-21  

Pharmacotherapeutic group

Other nervous system drugs

Therapeutic indication

Spinraza is indicated for the treatment of 5q Spinal Muscular Atrophy.

Assessment History

Changes since initial authorisation of medicine

Name Language First published Last updated

Initial marketing-authorisation documents

Name Language First published Last updated
Spinraza : EPAR - Public assessment report HR = Hrvatski 2017-06-21  
CHMP summary of positive opinion for Spinraza HR = Hrvatski 2017-04-21