MAIDENHEAD, England–(BUSINESS WIRE)–The National Institute for Health and Care Excellence (NICE) has
recommended funding for Spinraza (nusinersen) on the National Health
Service (NHS). The positive recommendation, subject to a five-year
managed access agreement (MAA), is for the treatment of infants,
children and adults with 5q spinal muscular atrophy (SMA), including
pre-symptomatic and SMA types I, II and III. SMA is a rare, debilitating
and life-threatening disease that results in severe progressive muscular
atrophy and weakness.1

Terry O’Regan, Vice President and Managing Director of Biogen UK and
Ireland, said:

“The NICE recommendation of nusinersen for infants, children, and
adults with 5q SMA, including pre-symptomatic, SMA types I, II and III,
is a momentous occasion for patients and their families who have fought
tirelessly for access to this life-changing medicine. This positive
outcome has been achieved through intensive and collaborative working
between the SMA community, NICE, NHS England, and Biogen. We will
continue to work with health authorities to ensure this welcome decision
translates into access as soon as possible for those awaiting treatment,
which includes providing NHS England with access to nusinersen for type
I patients immediately.”

NICE’s evaluation of nusinersen was based on the largest body of
clinical evidence currently available across all types of SMA, with six
years of data including more than 300 patients treated with
pre-symptomatic, infantile (type I) and later-onset (types II and III)
SMA.2 To date, more than 7,500 patients worldwide have been
treated with nusinersen, from infants to adults.2

The decision builds on Biogen’s commitment to find solutions to provide
broad access to innovative therapies through collaborating closely with
governments and communities around the world on new business models. In
Europe, a key component of that work is Biogen’s portfolio of
biosimilars — cost-saving biologic medicines that are similar to
currently available biologic therapies known as originators. Biosimilar
products are strategically important in creating scope in healthcare
budgets for patients to access innovative medicines, as Biogen works
with payers and health systems globally to achieve this goal. In Europe,
approximately 145,000 patients have been treated with a Biogen
biosimilar and, based on internal estimates, Biogen expects the uptake
to contribute an estimated healthcare savings of up to 1.8 billion Euros
in 2019.2


About Spinraza® (nusinersen)

Spinraza (nusinersen) is licensed globally for the treatment of 5q SMA.
Currently, patients in 24 European countries (and many more around the
world) have access to nusinersen via regular reimbursement, with most
countries making nusinersen available to treat a broad range of patients.3

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.4 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, which delivers therapies directly into
the cerebrospinal fluid (CSF) around the spinal cord, where motor
neurons degenerate in individuals with SMA due to insufficient levels of
SMN protein.

Nusinersen must be administered via intrathecal injection, which
delivers therapies directly to the cerebrospinal fluid (CSF) around the
spinal cord,5 where motor neurons degenerate in individuals
with SMA due to insufficient levels of SMN protein.6

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.5 Additionally, infants treated with
nusinersen demonstrated a statistically significant reduction in the
risk of death or permanent ventilation compared to sham.5

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.5
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. 5

More about Spinal Muscular Atrophy (SMA)

At any one time, it is thought that there are between 650 and 1,300
children and adults in the UK living with SMA (all types).7

Whilst type I SMA (infantile onset) develops in babies less than six
months old and can typically lead to death within the first few years of
life, type II appears in infants who are 7-18 months old.8
Type III develops after 18 months of age and type IV begins in adulthood.8

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
as well as related therapeutic adjacencies. 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. Today, Biogen has the leading portfolio of
medicines to treat multiple sclerosis (MS) and is also focused on
advancing research programmes in spinal muscular atrophy, advancing
neuroscience research programmes in MS and neuroimmunology, Alzheimer’s
disease and dementia, movement disorders, neuromuscular disorders, acute
neurology, neurocognitive disorders, pain and ophthalmology. Biogen is
one of only a handful of companies with the deep scientific expertise
needed to produce biosimilars of advanced biologics. To learn more,
please visit:

We routinely post information that may be important to investors on our
website at
To learn more, please visit and
follow us on social media – TwitterLinkedInFacebookYouTube.


1. 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.

2. Biogen. Data on File.

3. Biogen. SMA Community Update. January 2019.

4. 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.

5. SPINRAZA Summary of Product Characteristics (SmPC).

6. Lunn MR, Wang CH. Spinal muscular atrophy. Lancet.

7. Spinal Muscular Atrophy UK, What is SMA?. Available at:
Last accessed: May 2019.

8. NHS Choices. Spinal Muscular Atrophy. Available at:
Last accessed: May 2019.


May 2019


David Robertson
Director, Corporate Communications, UK
and Ireland
+44 (0) 1628 512726
[email protected]