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Spinraza®â–¼(nusinersen) yet to be made available by NHS England following NICE interim rejection


Maidenhead, UK. – 16 November, 2018Biogen was announced as the winner of the Orphan Product Award at last night’s UK Prix Galien 2018, for Spinraza®, which in 2017 became the first and only approved treatment for 5q spinal muscular atrophy (SMA). This is further recognition of the innovative science behind the development of nusinersen, which has already won five Prix Galien awards in the U.S, Italy, the Netherlands, Belgium-Luxembourg and Germany. In addition, the scientists behind nusinersen won the Breakthrough Prize, which honours transformative advances toward understanding living systems and extending human life, earlier this month.


Prix Galien was introduced to recognise innovative and important medicines. The awards are judged by prominent healthcare leaders, academics and specialists. It is one of life science’s most prestigious awards for innovation, recognising breakthrough therapies which improve the human condition and address unmet need. The ceremony was held at the House of Commons, London on 15th November 2018, where nusinersen won by unanimous decision, having been one of seven finalists in the Orphan Product category.


Nusinersen gained European marketing authorisation in May 2017 following accelerated assessment, and a licence was given based on the strength of the clinical trial interim results. The medicine is now available to patients with SMA in 22 European countries (including Scotland for Type 1) and many more across the world,1 but not in England, Wales and Northern Ireland following a ‘minded no’ issued by NICE in August 2018. SMA is a rare muscle wasting condition which affects a person’s ability to walk, eat and ultimately breathe.2 The leading cause of infant genetic death,3 those with the most severe form of SMA are unlikely to live to see their second birthday.4


“Biogen is delighted to have been recognised by the prestigious Prix Galien here in the UK with the Orphan Product Award for nusinersen,” said Terry O’Regan, Vice President and Managing Director, UK and Ireland at Biogen. “Nusinersen is the first approved medicine to treat spinal muscular atrophy, a serious and life-threatening disease that impacts patients and families severely, and we are humbled by the opportunity to change this for patients in the UK. We are committed to working with NICE and NHSE to swiftly reach a decision to bring this important treatment to all who may benefit and bring equality to patients in England, Wales and Northern Ireland, so that they can have the same access as those in countries such as Germany, Italy, Romania, Greece and Croatia.”


SMA occurs when a person has mutations or deletions in the Survival Motor Neuron 1 (SMN1) gene, causing issues with nerve functionality. We all have a backup of this gene, called SMN2, however it does not produce enough SMN protein on its own. Nusinersen works by increasing the amount of full-length SMN protein,5 with the potential to fundamentally change the course of the disease in individuals with SMA.


O’Regan continued: “We are thrilled by the recognition of nusinersen’s clinical value, however we want this to translate into availability for the patients who desperately need it. The UK has a much lower and slower uptake of medicines for rare diseases with only 50% of approved rare diseases medicines making it to patients in England, and those that do take two years on average.6 Biogen is committed to working with all stakeholders to change this in the future.”




About Spinraza® (nusinersen)

Spinraza (nusinersen) is licensed globally for the treatment of 5q SMA. Currently, patients in 22 European countries have access to nusinersen via regular reimbursement, with most countries making nusinersen available to treat a range of patients.1 In the UK, NICE appraisal of nusinersen is currently ongoing and Biogen continues to actively collaborate with patients, clinicians and government bodies to make this important treatment available to all who may benefit.


Nusinersen is an antisense oligonucleotide (ASO), using Ionis’ proprietary antisense technology that is designed to treat SMA caused by mutations or deletions in the Survival Motor Neuron 1 (SMN1) gene located in chromosome 5q that leads to SMN protein deficiency. Nusinersen alters the splicing of SMN2 pre-mRNA in order to increase production of full-length SMN protein.5 ASOs are short synthetic strings of nucleotides designed to selectively bind to target RNA and regulate gene expression. Through use of this technology, nusinersen has the potential to increase the amount of full-length SMN protein in individuals with SMA.


Nusinersen must be administered via intrathecal injection, which delivers therapies directly to the cerebrospinal fluid (CSF) around the spinal cord,7 where motor neurons degenerate in individuals with SMA due to insufficient levels of SMN protein.8


Following the assessment of nusinersen in patients affected by SMA, results from the ENDEAR end of study analysis indicate that some infants achieved motor milestones including full head control, ability to roll, sit, and stand.7 Additionally, infants treated with nusinersen demonstrated a statistically significant reduction in the risk of death or permanent ventilation compared to sham.7


Nusinersen demonstrated a favourable benefit-risk profile. The most common adverse events were headache, vomiting and back pain, considered related to the lumbar puncture procedure. The incidence and severity of these events were consistent with events expected to occur with lumbar puncture. Thrombocytopenia and coagulation abnormalities, including acute severe thrombocytopenia, and renal toxicity have been observed after administration of some ASOs, however this was not demonstrated in the clinical trials of nusinersen. The primary route of elimination is expected via urinary excretion of nusinersen and its metabolites.7 Adverse reactions and complications including serious infection, such as meningitis, may occur as part of performing the lumbar puncture procedure. Hydrocephalus, not related to meningitis or bleeding, has been reported in patients treated with nusinersen, however causality has not been demonstrated.


About Biogen
At Biogen, our mission is clear: we are pioneers in neuroscience. Biogen discovers, develops, and delivers worldwide innovative therapies for people living with serious neurological and neurodegenerative diseases. One of the world’s first global biotechnology companies, Biogen was founded in 1978 by Charles Weissmann, Heinz Schaller, Kenneth Murray and Nobel Prize winners Walter Gilbert and Phillip Sharp, and today has the leading portfolio of medicines to treat multiple sclerosis; has introduced the first and only approved treatment for spinal muscular atrophy; and is focused on advancing neuroscience research programmes in Alzheimer’s disease and dementia, multiple sclerosis and neuroimmunology, movement disorders, neuromuscular disorders, pain, ophthalmology, neuropsychiatry, and acute neurology. Biogen also manufactures and commercialises biosimilars of advanced biologics. To learn more, please visit



1 Biogen SMA community update, October 2018.

2 NHS Choices. Spinal Muscular Atrophy. Available at: Last accessed: November 2018.

3 Committee for Medicinal Products for Human Use (CHMP), Assessment report – Spinraza, International non-proprietary name: nusinersen, 21 April 2017. Procedure No. EMEA/H/C/004312/0000.

4 The SMA Trust, About SMA. Available at: Last accessed: November 2018.

5 Hua Y, Sahashi K, Hung G, Rigo F, Passini MA, Bennett CF, Krainer AR. Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model. Genes Dev. 2010;24(15):16344-44.

6 Zamora B, Maignen F, O’Neill P, Mestre-Ferrandiz J, Garau M. Comparing Access to Orphan Medicinal Products (OMPs) in the United Kingdom and other European countries. Office of Health Economics Consulting Report. March 2017.

7 Spinraza Summary of Product Characteristics (SmPC).

8 Lunn MR, Wang CH. Spinal muscular atrophy. Lancet. 2008;371(9630):2120-2133.


â–¼ This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions.



November 2018

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Last Updated: 16-Nov-2018