- Treatment with SPINRAZA™ resulted in survival of 100 percent of
infants and none requiring permanent ventilation
- Recently published outcomes include motor milestones achieved
in 100 percent of infants sitting without support and 88 percent
walking independently
MISSISSAUGA, ON, Oct. 3, 2019 /CNW/ - Biogen Canada is pleased to
announce results from the NURTURE study, which demonstrated that
infants who initiated treatment with SPINRAZA™ (nusinersen
injection) prior to the onset of clinical symptoms of spinal
muscular atrophy (SMA) attained positive results compared to the
natural history of the disease. These results have been published
in the journal Neuromuscular Disorders. NURTURE is the first
study investigating a treatment targeting the underlying cause of
spinal muscular atrophy (SMA) in infants treated
pre-symptomatically.
NURTURE is an ongoing, Phase 2, open-label study of 25 infants
with the genetic diagnosis of SMA (most likely to develop SMA Type
1 or 2) who received their first dose of SPINRAZA™ in the
pre-symptomatic stage and before six weeks of age. The study,
conducted at 15 sites in seven countries, has close to four years
of data (up to 45.4 months). When compared with the natural history
of the disease, the results are dramatic in their impact on
changing the course of SMA. As of March
2019, all participants were alive, without the need for
permanent ventilation and experienced continuous improvements, with
the majority achieving motor milestones in timelines consistent
with normal development. The results also demonstrated durability
of effect with children making progress throughout the
study.
"By showing dramatic clinical benefit in very young SMA
patients, the NURTURE study throws into sharp relief the new
therapeutic SMA era the world has entered," said Ottawa pediatric endocrinologist and SMA
newborn screening advocate, Dr. Alex
MacKenzie. "The results make a compelling case for the
pre-symptomatic treatment of SMA, underlining the critical
importance of newborn screening for all Canadian infants."
As of March 2019, all infants in
the study were 25 months or older, past the typical age of symptom
onset for SMA Type 1 and Type 2 and were alive without the need for
permanent ventilation. In comparison to the natural history of SMA,
many of these infants would likely have passed away or require
permanent ventilation on average by 13.5 months. In both the
children with two and three copies of SMN2, treatment with
SPINRAZA™ demonstrated rapid onset of improvement and durability of
effect with their mean Children's Hospital of Pennsylvania Infant
Test of Neuromuscular Disorders (CHOP-INTEND) score of motor
function reaching the maximum mean score of 64 for all participants
with three copies of SMN2 (n=10) and a mean of 62.1 for those with
two copies of SMN2 (N=15).
"We are pleased to share this very exciting data with the SMA
community in Canada because it
adds to our understanding of what optimal disease management with
SPINRAZA™ could be with young patients with SMA," said Marina Vasiliou, Vice President and Managing
Director of Biogen Canada. "Ultimately what we want for patients is
to change the course of their disease which is why we are committed
to ongoing research in SMA. This study, and others we are
conducting are part of Biogen's commitment to helping Canadians
affected by SMA."
Reaction from the SMA community
Susi Vander Wyk, Executive
Director, Cure SMA Canada: "SPINRAZA™ is a life-changing
medicine and these eye-opening results in infants with SMA has
brought hope for the future that these patients never had. SMA
babies deserve a chance to meet motor milestones and to function
independently as long as they can."
Barbara Stead-Coyle, CEO,
Muscular Dystrophy Canada: "We welcome the growing body of
evidence with SPINRAZA™ and believe that research like this is
critical for the SMA community. With this unparalleled evidence in
newborn infants, it is critical that newborn screening for SMA gets
adopted in every province in Canada. NURTURE is evidence that shows the
sooner the infant is treated, the better the outcomes for the
infant and the family."
The Health Canada approval of SPINRAZA™ in June 2017 for pre-symptomatic, Type I, II and III
SMA patients regardless of age is based on Biogen's extensive
clinical development program for this treatment, including data
from over 300 patients across a broad range of SMA populations.
Biogen continues to partner with the medical community in
Canada and worldwide for the
ongoing evaluation of the effectiveness and tolerability of this
treatment in SMA.
About Spinal Muscular Atrophy (SMA)
SMA is a rare, debilitating neurodegenerative condition that is
characterized by loss of motor neurons in the spinal cord and lower
brain stem, resulting in severe and progressive muscular atrophy
and weakness.
SMA is the leading genetic cause of death among infants. It is
estimated that 1 in 10,000 live births are affected by SMA.
Untreated, children with the most severe form of SMA (Type 1 or
infantile-onset SMA) rarely live to see their second birthday.
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. Ultimately, individuals with SMA can become
paralyzed and have difficulty performing the basic functions of
life, like breathing and swallowing.
People with Type I 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 typically live
beyond two years without respiratory support. People with Type II
and Type III SMA produce greater amounts of SMN protein and have
less severe, but still life-altering forms of SMA.
About SPINRAZA™ (nusinersen injection)
SPINRAZA™ (nusinersen injection) is the first and only approved
treatment in Canada for spinal
muscular atrophy (SMA). It is the only SMA therapy with a sustained
efficacy and manageable safety profile and is supported by the
largest clinical data set and real-world evidence, with more than
8,400 patients treated worldwide for up to nearly six years.
Approved in more than 44 countries, it has been shown to improve
survival for those with the most severe form of SMA (Type 1 or
infantile-onset SMA) and has enabled children, teens and adults
with SMA to maintain or improve motor function, transforming the
course of the disease.
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, and today has the
leading portfolio of medicines to treat multiple sclerosis, has
introduced the first approved treatment for spinal muscular
atrophy, and is focused on advancing neuroscience research programs
in MS 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.ca . To learn more, please visit
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SOURCE Biogen Canada