- Access to the first and only approved treatment for
pre-symptomatic, Type I, II, and III SMA patients under the age of
18
- Ontario joins Saskatchewan and Quebec to give patients with SMA broader
access to SPINRAZA™
MISSISSAUGA, ON, June 13, 2019 /CNW/ - Biogen Canada is pleased to
announce that patients in Ontario
living with spinal muscular atrophy (SMA) now have broader publicly
funded access to SPINRAZA™ (nusinersen), the first and only
treatment approved in Canada for
SMA.
This announcement follows the decision in November 2018 to provide publicly funded access
for those living with Type I SMA. Ontario has expanded coverage for SPINRAZA™ to
include the following patients:
- Patients who are pre-symptomatic with two or three copies of
the survival motor neuron 2 (SMN2) gene;
- Patients with disease duration of less than six months, two
copies of the SMN2 gene, and symptom onset after the first week
after birth and on or before seven months of age;
- Patients under the age of 18 with symptom onset after six
months of age, who have never achieved the ability to walk
independently;
- Other Type II and III patients regardless of ever achieving the
ability to walk independently are encouraged to apply for access
through their clinicians to be considered on a case by case
basis;
- Treatment for adult patients can be approved exceptionally on a
case by case basis.
"We welcome the decision by the Ontario government to grant broader access to
SPINRAZA™," said Marina Vasiliou,
Vice President and Managing Director of Biogen Canada. "We believe
that all SMA patients, including adults, should have broad access
and we will continue to work with all jurisdictions until this is
achieved in Canada."
The Ontario government decision
follows the decisions of Quebec,
Saskatchewan, Non-Insured Health
Benefits (NIHB) and more than 44 countries around the world who
grant broad reimbursement to SPINRAZA™.
The Health Canada approval of SPINRAZA™ in June 2017 for all pre-symptomatic, type I, II and
III SMA patients regardless of age is based on Biogen's extensive
clinical development program for this treatment. Biogen continues
to partner with the medical community in Canada and worldwide for the ongoing
evaluation of the long-term effectiveness of this treatment in
SMA.
Reaction from the Canadian SMA community
Toronto pediatric
neurologist Dr. Jiri Vajsar welcomes the news for young SMA
patients: "This is very good news. We feel that broad access to
this disease-modifying therapy for SMA is critical and will give
more patients and families dealing with the disease hope to slow
the natural progression of the disease and improve or at least
maintain their motor control, strength, function and
independence. This is ultimately what we aspire to achieve
for all SMA patients across the spectrum of the disease."
London pediatric
neurologist, Dr. Craig Campbell is
optimistic about the Ontario
government decision: "The decision to expand nusinersen access
in Ontario is an important,
thoughtful and positive decision for the SMA community. It will
allow a wide range of people with SMA to realize the substantial
benefits of this medication. As an SMA community this allows us to
make sure nusinersen gets to those who need it now, and
collectively document the long-term real world data to further
demonstrate the positive impact of nusinersen."
Toronto neurologist Dr.
Aaron Izenberg recognizes the
significance of what this means for adults living with SMA:
"SPINRAZA™ is currently the only approved disease-modifying
treatment for SMA. I am pleased with the Ontario government's decision to include
access for adults on a case by case basis."
Susi Vander Wyk, Executive
Director, Cure SMA Canada: "We have been hoping this day
would come and are thrilled that the Ontario government has recognized the need for
SPINRAZA™ to be accessible more broadly. We welcome this step
forward and urge all other jurisdictions to ensure this needed
treatment is readily accessible to all patients across Canada who can benefit."
Barbara Stead-Coyle, CEO,
Muscular Dystrophy Canada: "SMA is a devastating and
debilitating disease for the individual and the families. We
are pleased with the Ontario
government's decision and will continue our work to advocate for
expanded access for all patients living with SMA, as the evidence
supporting SPINRAZA™ shows that treatment can have a meaningful
impact across all forms of the disease."
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 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 (infantile-onset) 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)
SPINRAZA™ (nusinersen) is the first and only approved treatment
in Canada for spinal muscular
atrophy (SMA). It is the only SMA therapy with a
well-established safety and durability profile and is supported by
the largest clinical data set and real-world evidence, with more
than 7,500 patients treated worldwide. Approved in more than
44 countries, it has been shown to improve survival for those with
the most severe form of SMA (infantile-onset) 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
www.biogen.ca and follow us on social media – Twitter,
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SOURCE Biogen Canada