The National Institute for Health and Care Excellence (NICE) have announced that the managed access agreement for Biogen's spinal muscular atrophy medicine nusinersen, brand name Spinraza▼, can now be used in paediatric patients who have recently lost the ability to walk independently.
This replaces the previous criteria that patients, who had previously gained ambulation, should still be able to walk independently at the start of their treatment.
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About Spinraza (source EMA)
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.
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.
Spinraza was first made available in the EU in 2017. It is manufactured by Biogen.
European Medicines Agency website page for Spinraza
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© NICE  Spinraza access agreement extended Available from: See Link below. All rights reserved. Subject to Notice of rights
NICE guidance is prepared for the National Health Service in England. All NICE guidance is subject to regular review and may be updated or withdrawn. NICE accepts no responsibility for the use of its content in this product/publication.
The information provided by NICE was accurate at the time this article was issued.
NICE information on Spinraza
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