Biogen Inc. (Nasdaq: BIIB) today announced that The National
Institute for Health and Care Excellence (NICE) in the United
Kingdom has recommended funding for SPINRAZA (nusinersen) on the
National Health Service (NHS). The positive recommendation is for
the treatment of infants, children and adults with 5q spinal
muscular atrophy (SMA), including pre-symptomatic and symptomatic
SMA Types 1, 2 and 3. SMA is a rare, debilitating and
life-threatening disease that results in severe, progressive
muscular atrophy and weakness.
“We applaud the decision by NICE to recommend
funding for SPINRAZA in the United Kingdom. This is a momentous
occasion for patients and their families and the result of a strong
collaboration between Biogen, NICE, NHS and the SMA community,”
said Chirfi Guindo, Executive Vice President, Global Product
Strategy and Commercialization at Biogen. “We are committed to
working with authorities to find solutions to fund innovation and
provide broad patient access through value-based contracting
programs and by enabling governments to leverage savings created by
our biosimilars portfolio.”
Established in 1999, NICE provides advice and
standards on value in healthcare to the NHS. The NICE
recommendation was based on the comprehensive set of data for
nusinersen highlighting its clinically meaningful benefits for
individuals in all age groups with SMA. New data on the efficacy
and safety of nusinersen was presented at the 71st Annual
Meeting of the American Academy of Neurology in Philadelphia (May
4-10). SPINRAZA is an antisense oligonucleotide (ASO) that targets
the underlying cause of the disease in order to increase production
of full-length survival motor neuron protein.
The decision builds on Biogen’s commitment to find
solutions to provide broad access to innovative therapies by
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 — biologic medicines that
are similar to currently available biologic therapies known as
originators. Biosimilar products benefit patients and are
strategically important as Biogen works with payers and health
systems globally with the goal of creating room in healthcare
budgets to provide access for patients to innovative therapies. 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.
About
SPINRAZA® (nusinersen)1-4SPINRAZA is the first
approved medicine for the treatment of spinal muscular atrophy
(SMA) and is currently available in more than 40 countries. As of
March 31, 2019, more than 7,500 individuals with SMA are being
treated with SPINRAZA worldwide, based on patients across the
post-marketing setting, Expanded Access Program (EAP) and clinical
trial participants.
SPINRAZA is an antisense oligonucleotide (ASO)
developed using Ionis’ proprietary antisense technology that is
designed to treat the root cause of SMA. SPINRAZA alters the
splicing of SMN2 pre-mRNA in order to increase production of
full-length spinal motor neuron (SMN) protein. ASOs are short
synthetic strings of nucleotides designed to selectively bind to
target RNA and regulate gene expression. Through use of this
technology, SPINRAZA has been shown to increase the amount of
full-length SMN protein in individuals with SMA. SPINRAZA is
administered via intrathecal injection, 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.
In the clinical trial program, SPINRAZA
demonstrated a favorable benefit-risk profile. The most common
adverse reactions that occurred in the SPINRAZA group were
respiratory infection and constipation. Serious adverse reactions
of atelectasis were more frequent in SPINRAZA-treated patients.
Coagulation abnormalities and thrombocytopenia, including acute
severe thrombocytopenia, have been observed after administration of
some ASOs. Individuals may be at increased risk of bleeding
complications. Renal toxicity has been observed after
administration of some ASOs. SPINRAZA is present in and excreted by
the kidney.
Biogen licensed the global rights to develop,
manufacture and commercialize SPINRAZA from Ionis Pharmaceuticals,
Inc. (Nasdaq: IONS), a leader in antisense therapeutics. Biogen and
Ionis conducted an innovative clinical development program, the
largest of its kind in SMA, that moved SPINRAZA from its first dose
in humans in 2011 to its first regulatory approval in five
years.
About SMA2,5 SMA is a rare,
genetic, neuromuscular disease that is characterized by loss of
motor neurons in the spinal cord and lower brain stem, resulting in
severe and progressive muscle atrophy and weakness. About 1 in
10,000 live births have a diagnosis of SMA. Ultimately, individuals
with SMA can lose the ability to walk and have difficulty
performing the basic functions of life, such as breathing and
swallowing, which results in significant healthcare intervention
and caregiver assistance. Left untreated, the majority of infants
with the most severe form of the disease (SMA Type 1) do not live
beyond their second birthday without respiratory intervention.
People with childhood or adult onset SMA (Type 2 or 3) produce
greater amounts of SMN protein resulting in less severe, but still
life-altering forms of the disease.
Due to a deletion of, or mutation in, the SMN1
gene, people with SMA do not produce enough SMN protein, which is
critical for the maintenance of motor neurons. The severity of SMA
correlates with the amount of SMN protein an individual has. People
with SMA Type 1, the form that requires the most intensive and
supportive care, produce very little SMN protein and do not achieve
the ability to sit without support or typically live beyond two
years without respiratory support. People with SMA Type 2 and Type
3 produce greater amounts of SMN protein and have less severe, but
still life-altering forms of SMA.
About BiogenAt 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, 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 programs in
multiple sclerosis and neuroimmunology, Alzheimer’s disease and
dementia, movement disorders, neuromuscular disorders, acute
neurology, neurocognitive disorders, pain and ophthalmology. Biogen
also commercializes biosimilars of advanced biologics.
We routinely post information that may be important
to investors on our website at www.biogen.com. To learn more,
please visit www.biogen.com and follow us on social media
– Twitter, LinkedIn, Facebook, YouTube.
Biogen Safe Harbor This news
release contains forward-looking statements, including statements
made pursuant to the safe harbor provisions of the Private
Securities Litigation Reform Act of 1995, about the potential
benefits, safety and efficacy of SPINRAZA; the results of certain
real-world data; the status of current regulatory filings; and the
potential of our commercial business, including SPINRAZA and
our biosimilars portfolio. These statements may be identified by
words such as “aim,” “anticipate,” “believe,” “could,” “estimate,”
“expect,” “forecast,” “goal,” “intend,” “may,” “plan,” “possible,”
“potential,” “will,” “would” and other words and terms of similar
meaning. Drug development and commercialization involve a high
degree of risk, and only a small number of research and development
programs result in commercialization of a product. Results in early
stage clinical trials may not be indicative of full results or
results from later stage or larger scale clinical trials and do not
ensure regulatory approval. You should not place undue reliance on
these statements or the scientific data presented.
These statements involve risks and uncertainties
that could cause actual results to differ materially from those
reflected in such statements, including without limitation
uncertainty of success in commercialization of SPINRAZA, which may
be impacted by, among other things, the level of preparedness of
healthcare providers to treat patients, difficulties in obtaining
or changes in the availability of reimbursement for SPINRAZA, the
effectiveness of sales and marketing efforts, problems with the
manufacturing process for SPINRAZA, the occurrence of adverse
safety events and/or unexpected concerns that may arise from
additional data or analysis; failure to obtain regulatory approvals
in other jurisdictions; risks of unexpected costs or delays;
failure to protect and enforce our data, intellectual property and
other proprietary rights and uncertainties relating to intellectual
property claims and challenges; risks related to our dependence on
third parties for the development and commercialization of
biosimilars; risks of legal actions, regulatory scrutiny or other
challenges to biosimilars; product liability claims; and third
party collaboration risks. The foregoing sets forth many, but not
all, of the factors that could cause actual results to differ from
our expectations in any forward-looking statement. Investors should
consider this cautionary statement, as well as the risk factors
identified in our most recent annual or quarterly report and in
other reports we have filed with the Securities and Exchange
Commission. These statements are based on our current beliefs and
expectations and speak only as of the date of this news release. We
do not undertake any obligation to publicly update any
forward-looking statements, whether as a result of new information,
future developments or otherwise.
Reference:* As of March 31, 2019, more than 7,500
individuals with SMA are being treated with SPINRAZA worldwide,
based on patients across the post-marketing setting, Expanded
Access Program (EAP) and clinical trial participants.
1. 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 Aug 1; 24(15):16344-44.
2. Finkel R, Chiriboga C, Vajsar J, et al.
Treatment of infantile-onset spinal muscular atrophy with
nusinersen: a phase 2, open-label, dose-escalation study. Lancet.
2016;388(10063):3017-3026.
3. Evers MM, Toonen LJ, van Roon-Mom WM. Antisense
oligonucleotides in therapy for neurodegenerative disorders. Adv
Drug Deliv Rev. 2015;87:90-103.
4. Lunn MR, Wang CH. Spinal muscular atrophy.
Lancet. 2008;371(9630):2120-2133.
5. Darras B, Markowitz J, Monani U, De Vivo D.
Chapter 8 - Spinal Muscular Atrophies. In: Vivo BTD, ed.
Neuromuscular Disorders of Infancy, Childhood, and Adolescence
(Second Edition). San Diego: Academic Press; 2015:117-145.
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