European Medicines Agency Accepts Tofersen Marketing Authorization
Application to Treat Rare, Genetic Form of ALS
Biogen Inc. (Nasdaq: BIIB) announced the European Medicines Agency
(EMA) has accepted the Marketing Authorization Application (MAA)
for review of tofersen, an investigational drug for the treatment
of superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis
(ALS). SOD1-ALS is a progressive and uniformly fatal disease that
affects less than 1,000 people in Europe.2 There is currently no
treatment targeted for SOD1-ALS.3
“Through our clinical development program, we have seen that
tofersen has the potential to slow the progression of this
relentless and ultimately fatal disease,” said Priya Singhal, M.D.,
M.P.H., Head of Global Safety and Regulatory Sciences and Interim
Head of R&D at Biogen. “Regulatory submissions in the U.S. and
now EU represent an important step in our efforts to bring the
first genetically-targeted treatment for SOD1-ALS to the ALS
community as quickly as possible.”
The MAA includes results from the Phase 3 VALOR study, its open
label extension (OLE) study, a Phase 1 study in healthy volunteers
and a Phase 1/2 study evaluating ascending dose levels. Also
included are the most current 12-month integrated results from
VALOR and the OLE study that were recently published in The New
England Journal of Medicine.
“The effects we have seen with tofersen diverge from the natural
history of the disease and bring hope for the treatment of
SOD1-ALS,” said Philip Van Damme, M.D., Ph.D., professor of
neurology and director of the Neuromuscular Reference Center at the
University Hospital Leuven in Belgium. “Today’s announcement is an
important milestone for the ALS community in Europe where there is
a tremendous need for additional treatment options.”
Tofersen is also under review with the U.S. Food and Drug
Administration with Priority Review and has a Prescription Drug
User Fee Act action date of April 25, 2023.
Biogen will maintain its early access program for tofersen,
which is now available in 34 countries. Biogen continues to
actively engage with other regulators around the world and will
provide updates when appropriate.
About TofersenTofersen is an antisense drug
being evaluated as a treatment of SOD1-ALS. Tofersen is designed to
bind to SOD1 mRNA, inducing its degradation by RNase-H to reduce
SOD1 protein production. In addition to the ongoing open label
extension of VALOR, tofersen is being studied in the Phase 3 ATLAS
study designed to evaluate whether tofersen can delay clinical
onset when initiated in presymptomatic individuals with a SOD1
genetic mutation and biomarker evidence of disease activity. Biogen
licensed tofersen from Ionis Pharmaceuticals, Inc. under a
collaborative development and license agreement.
About Amyotrophic Lateral Sclerosis and
SOD1-ALSAmyotrophic lateral sclerosis (ALS) is a rare,
progressive and fatal neurodegenerative disease that results in the
loss of motor neurons in the brain and the spinal cord that are
responsible for controlling voluntary muscle movement. People with
ALS experience muscle weakness and atrophy, causing them to lose
independence as they steadily lose the ability to move, speak, eat,
and eventually breathe. Average life expectancy for people with ALS
is three to five years from time of symptom onset.3
Multiple genes have been implicated in ALS. Genetic testing
helps determine if a person’s ALS is associated with a genetic
mutation, even in individuals without a family history of the
disease. Currently, there are no genetically targeted treatment
options for ALS. Mutations in the SOD1 gene are responsible for
approximately 2 percent of the estimated 168,000 people who have
ALS globally (SOD1-ALS).2 Life expectancy in SOD1-ALS varies
widely with some patients surviving less than a year.4
Biogen’s Continuous Commitment to ALSFor over a
decade, Biogen has been committed to advancing ALS research to
provide a deeper understanding of all forms of the disease. The
company has continued to invest in and pioneer research despite
making the difficult decision to discontinue a late-stage ALS asset
in 2013. Biogen has applied important learnings to its portfolio of
assets for genetic and other forms of ALS, with the goal of
increasing the probability of bringing a potential therapy to
patients in need. These applied learnings include evaluating
genetically validated targets in defined patient populations,
pursuing the most appropriate modality for each target and
employing sensitive clinical endpoints. Today, the company has a
pipeline of investigational drugs being evaluated in ALS, including
tofersen and BIIB105.
About BiogenAs pioneers in neuroscience, Biogen
discovers, develops, and delivers worldwide innovative therapies
for people living with serious neurological 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, Sir Kenneth Murray, and Nobel Prize
winners Walter Gilbert and Phillip Sharp. Today, Biogen has a
leading portfolio of medicines to treat multiple sclerosis, has
introduced the first approved treatment for spinal muscular
atrophy, and developed the first and only approved treatment to
address a defining pathology of Alzheimer’s disease. Biogen is also
commercializing biosimilars and focusing on advancing one of the
industry’s most diversified pipelines in neuroscience that will
transform the standard of care for patients in several areas of
high unmet need.
In 2020, Biogen launched a bold 20-year, $250 million initiative
to address the deeply interrelated issues of climate, health, and
equity. Healthy Climate, Healthy Lives™ aims to eliminate fossil
fuels across the company’s operations, build collaborations with
renowned institutions to advance the science to improve human
health outcomes, and support underserved communities.
We routinely post information that may be important to investors
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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, including statements about results
from the Phase 3 VALOR study of tofersen or its OLE; the potential
clinical effects of tofersen; the potential benefits, safety and
efficacy of tofersen; the clinical development program for
tofersen; the potential approval of tofersen; the identification
and treatment of ALS; our research and development program for the
treatment of ALS; the potential of our commercial business and
pipeline programs, including tofersen; and risks and uncertainties
associated with drug development and commercialization. These
forward-looking statements may be accompanied by words such as
“aim,” “anticipate,” “believe,” “could,” “estimate,” “expect,”
“forecast,” “intend,” “may,” “plan,” “potential,” “possible,”
“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 the development and potential commercialization of
tofersen; the risk that we may not fully enroll our clinical trials
or enrollment will take longer than expected; unexpected concerns
may arise from additional data, analysis or results obtained during
our clinical trials; regulatory authorities may require additional
information or further studies, or may fail or refuse to approve or
may delay approval of our drug candidates, including tofersen; the
occurrence of adverse safety events; the risks of unexpected
hurdles, 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; product liability claims; and the direct and indirect
impacts of the ongoing COVID-19 pandemic on our business, results
of operations and financial condition. 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 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.
References:
- Trist BG, et al. Altered SOD1
maturation and post-translational modification in amyotrophic
lateral sclerosis spinal cord. Brain. 2022 Sep 14;145(9):3108-3130.
doi: 10.1093/brain/awac165.
- Brown CA, Lally C, Kupelian V,
Flanders WD. Estimated Prevalence and Incidence of Amyotrophic
Lateral Sclerosis and SOD1 and C9orf72 Genetic Variants.
Neuroepidemiology. 2021;55(5):342-353. doi: 10.1159/000516752. Epub
2021 Jul 9.
- Brown RH, Al-Chalabi A. Amyotrophic
Lateral Sclerosis. N Engl J Med. 2017 Jul 13
- Bali T, et al. Defining SOD1 ALS natural history to guide
therapeutic clinical trial design. J Neurol Neurosurg Psychiatry.
2017 Feb;88(2):99-105. doi: 10.1136/jnnp-2016-313521. Epub 2016 Jun
3.
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