The National Institute for Health and Care Excellence (NICE) have announced that patients in England and Wales can now receive treatment with Biogen's spinal muscular atrophy treatment nusinersen, brand name Spinraza▼.
The approval comes after NICE, Biogen and the NHS agreed a price for the medicine.
Nusinersen is the first treatment that targets the underlying cause of SMA.
SMA affects the nerves in the spinal cord controlling movement. This causes muscle weakness, progressive loss of movement, and difficulty breathing and swallowing.
People with the most severe forms of SMA usually die before the age of 2. There are currently no active treatments targeting the underlying cause of SMA so the condition is managed through supportive care which aims to minimise the impact of disability, address complications and improve quality of life.
It is estimated there are there are between 600 and 1200 children and adults in the UK living with SMA.
NICE had previously not been able to recommend nusinersen for routine use because of uncertainties over its long-term effectiveness and its high cost.
You can follow developments on Spinraza by using our Medicines Tracker service which provides users with updates about the medicines they are interested in.
The announcement follows the agreement of a deal between NHS England and Biogen which will see the NHS fund treatment with nusinersen for a time-limited period, allowing further data to be collected on its effectiveness.
The treatment will be made available to the youngest and most severely affected (SMA type 1) patients immediately by Biogen, with NHS England offering funding on NICE’s publication of final guidance next month.
For older babies, children and young adults with less severe symptoms (SMA types 2 and 3), the NHS will begin to roll out nusinersen shortly after NICE’s guidance is published.
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 Idec.
European Medicines Agency website page for Spinraza
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NICE press release on Spinraza
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