International Prix Galien Recognizes SPINRAZA® as Best
Biotechnology Product
Biogen Inc. (Nasdaq: BIIB) has been awarded the 2018 International
Prix Galien as Best Biotechnology Product for SPINRAZA
(nusinersen), the first and only treatment for spinal muscular
atrophy (SMA). The prestigious honor marks the seventh Prix Galien
for SPINRAZA, following country recognitions in the U.S., Germany,
Italy, Belgium-Luxembourg, the Netherlands and the U.K. The
international award was presented at a ceremony in Dakar, Senegal
on November 28, 2018.
“We are honored that the Galien Foundation
continues to recognize the groundbreaking science and profound
clinical impact of SPINRAZA with this international award,” said
Michel Vounatsos, chief executive officer of Biogen. “The ability
to bring the first and only therapy to people living with
SMA is the result of a close collaboration with the
SMA community, academia, researchers and our colleagues at Ionis,
which has made a meaningful difference in this area of high unmet
medical need.”
The International Prix Galien is given every two
years by Prix Galien International Committee members in recognition
of excellence in scientific innovation to improve human health. To
qualify for International Prix Galien consideration, nominees must
have won a Prix Galien in an individual member country.
SMA is a progressive, debilitating neuromuscular
disease that impacts infants, children and adults. It affects
approximately one in every 10,000 live births and results in muscle
weakness which can take away a person’s ability to walk, eat and
ultimately breathe.1 The U.S. Food and Drug Administration (FDA)
approved SPINRAZA on December 23, 2016, under priority review for
the treatment of SMA in pediatric and adult patients. In June 2017
SPINRAZA was granted marketing authorization in the European Union
to treat individuals with 5q SMA, the most common form of the
disease representing approximately 95 percent of all SMA
cases.2
About The Galien Foundation The
Galien Foundation fosters, recognizes and rewards excellence in
scientific innovation to improve the state of human health. The
Foundation oversees and directs activities in the USA for the Prix
Galien, an international award that recognizes outstanding
achievements in improving the human condition through the
development of innovative therapies. The Prix Galien was created in
France in 1970 in honor of Galen, the father of medical science and
modern pharmacology.
SPINRAZA Program Status SPINRAZA
is the first and only approved medicine for the treatment of spinal
muscular atrophy (SMA) and is currently approved in the U.S., the
European Union, and Japan, among other countries. Biogen has
submitted regulatory filings in additional countries and plans to
initiate additional filings in other countries. As of September 30,
2018 nearly 6,000 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.
To support the urgent need for treatment for the
most severely affected individuals living with SMA, Biogen
initiated one of the largest, global, pre-approval EAPs in any rare
disease, providing access to therapy free of charge. From its
launch to June 30, 2018, the EAP has provided treatment access to
more than 750 patients across 29 countries. Biogen also supports a
Named Patient Sales Program (NPP), which allows access to SPINRAZA
in countries where it is not commercially available.
Biogen licensed the global rights to develop,
manufacture and commercialize SPINRAZA from Ionis Pharmaceuticals
(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 SMA1,3-6SMA 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. Ultimately,
individuals with SMA may lose the ability to walk and can 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 do not live to
see their second birthday without respiratory intervention.
Due to a deletion of, or mutation in, the SMN1
gene, people with SMA do not produce enough survival motor neuron
(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 Type 1 SMA, 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 live beyond two years without respiratory support.
People with Type 2 and Type 3 SMA produce greater amounts of SMN
protein and have less severe, but still life-altering forms of
SMA.
About SPINRAZA®
(nusinersen)7-10SPINRAZA is an antisense
oligonucleotide (ASO), developed using Ionis’ proprietary antisense
technology, that is designed to treat SMA caused by mutations or
deletions in the SMN1 gene, located in chromosome 5q, that leads to
a deficiency in SMN protein. SPINRAZA alters the splicing of SMN2
pre-mRNA in order to increase production of full-length 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 must be 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.
Please click for Important Safety Information and
full Prescribing Information in the U.S. and Europe, or visit your
respective country’s product website.
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.
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 Alzheimer’s disease and dementia, multiple
sclerosis and neuroimmunology, movement disorders, neuromuscular
disorders, pain, ophthalmology, neuropsychiatry and acute
neurology. Biogen also manufactures and 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 press
release contains forward-looking statements, including statements
made pursuant to the safe harbor provisions of the Private
Securities Litigation Reform Act of 1995, relating to the potential
benefits, safety and efficacy of SPINRAZA; the results of certain
real-world data; the status of our current regulatory filings; our
plans for additional regulatory filings in other jurisdictions;
planning and timing for commercial launch; and availability of
patient access and reimbursement pathways, which may vary on a
country-by-country basis. These forward-looking statements may be
accompanied by words such as “aim,” “anticipate,” “believe,”
“could,” “estimate,” “expect,” “forecast,” “goal,” “intend,” “may,”
“plan,” “possible,” “potential,” “will” 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 the 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; regulatory authorities
may require additional information or further studies, or may fail
or refuse to approve or may delay approval of SPINRAZA or expansion
of product labeling; the occurrence of adverse safety events; risks
of unexpected costs or delays; we may encounter other unexpected
hurdles; failure to protect and enforce our data, intellectual
property and other proprietary rights and uncertainties relating to
intellectual property claims and challenges; 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 U.S.
Securities and Exchange Commission. These statements are based on
our current beliefs and expectations and speak only as of the date
of this press 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.
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Chapter 8 - Spinal Muscular Atrophies. In: Vivo BTD, ed.
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2. Farrar. Genetics of spinal muscular atrophy:
progress and challenges. Neurotherapeutics. 2015
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3. Lefebvre S, Burglen L, Reboullet S, et al.
Identification and characterization of a spinal muscular
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4. Mailman MD, Heinz JW, Papp AC, et al. Molecular
analysis of spinal muscular atrophy and modification of the
phenotype by SMN2. Genet Med. 2002;4(1):20-26.
5. Monani UR, Lorson CL, Parsons DW, et al. A
single nucleotide difference that alters splicing patterns
distinguishes the SMA gene SMN1 from the copy gene SMN2. Hum Mol
Genet. 1999;8(7):1177-1183.
6. Peeters K, Chamova T, Jordanova A. Clinical and
genetic diversity of SMN1-negative proximal spinal muscular
atrophies. Brain.2014;137(Pt 11):2879-2896.
7. 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.
8. 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.
9. Evers MM, Toonen LJ, van Roon-Mom WM. Antisense
oligonucleotides in therapy for neurodegenerative disorders. Adv
Drug Deliv Rev. 2015;87:90-103.
10. Lunn MR, Wang CH. Spinal muscular atrophy.
Lancet. 2008;371(9630):2120-2133.
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