NEW YORK, May 24, 2019 /PRNewswire/ -- The Muscular
Dystrophy Association (MDA) today celebrated the decision by the
U.S. Food and Drug Administration (FDA) to grant approval of
Zolgensma (onasemnogene abeparvovac-xioi), the first gene therapy
for a neuromuscular disease. Zolgensma is a one-time intravenous
(into the vein) infusion for the treatment of pediatric
patients less than 2 years of age with spinal muscular atrophy
(SMA) with bi-allelic mutations in the survival motor neuron 1
(SMN1) gene, including those who are pre-symptomatic at
diagnosis. SMA is the leading genetic cause of infant death.
Zolgensma is designed to target the genetic root cause of SMA by
delivering the survival motor neuron gene (SMN), which is
missing or mutated in SMA. Zolgensma will be made available in
the United States and will be
marketed by AveXis, a Novartis company.
Approval of the therapy marks another historic achievement for
the SMA community. Now, in addition to Spinraza — the first SMA
disease-modifying therapy, which was approved in December 2016 for SMA — patients will have access
to another promising therapy.
For decades, MDA has funded research aimed at the discovery of
genes causing neuromuscular disease and has supported work to
develop therapies that address the root cause of disease. Zolgensma
is only the second gene therapy approved by the FDA to treat any
disease, placing the field of neuromuscular disease at the
forefront of genetic medicine.
"Zolgensma is poised to be another life-altering therapy for the
SMA community," says MDA President and CEO Lynn O'Connor Vos. "It represents a breakthrough
toward the promise of safe and effective gene therapies, and it may
catalyze the development of other gene therapies to treat a range
of rare neuromuscular diseases."
Zolgensma may be the first of many gene therapies targeting the
genetic root cause of disease, highlighting the importance of years
of investment by MDA and others into gene identification and
research unlocking the cause of disease. In addition, MDA has
funded landmark research focused on developing and refining gene
delivery tools and has supported the work to establish protocols
for safe and effective gene therapy clinical trials. While this may
be the first gene therapy for treating neuromuscular disease, the
increasing pace of drug development holds immense promise for the
future of the field. In the past decade alone, seven therapies for
treating neuromuscular diseases have been approved by the FDA.
Clinical trials of Zolgensma deliver on the promise of gene
therapy
Positive results of a pivotal phase 1 START trial showed that
babies who received Zolgensma lived longer compared to the normal
course of the disease, and they achieved and maintained motor
milestones that infants with SMA type 1 normally would not be
expected to achieve, like holding their heads erect and sitting,
standing, or walking without support. Patients observed in a
long-term follow-up trial to the phase 1 study continued to gain
strength and achieve new milestones; two additional patients were
able to stand and walk alone without assistance at the end of the
follow-up period.
Interim data from the phase 3 STR1VE trial presented by AveXis
at the 2019 MDA Clinical & Scientific Conference in April and
the 2019 Academy of Neurology Annual Meeting in May validated the
pilot trial results. In this study, infants treated with Zolgensma
showed prolonged survival, increases in motor function, and
achievement of motor milestones beyond what would be expected for
infants with SMA type 1. AveXis is currently testing Zolgensma in
clinical trials for patients with SMA type 2 as well as in
pre-symptomatic SMA patients.
Zolgensma was also found to be well tolerated and safe in the
more than 150 patients who have been treated to date. The most
commonly observed adverse events were elevated liver enzymes and
vomiting. Transient decreases in platelet counts (platelets are
blood cells that play an important role in preventing bleeding)
were observed at different time points after Zolgensma infusion.
Zolgensma uses the adeno-associated viral vector 9 (AAV9) to
deliver the missing SMN gene. Because AAV9 is a naturally
occurring virus, some children may have antibodies against this
virus, making them ineligible for treatment. However, out of the
177 patients who underwent pre-treatment screening, only 5% were
unable to receive treatment because of high levels of antibodies
against the viral vector that delivers the gene product.
About OneGene Program
OneGene Program, AveXis' patient support program, provides a
dedicated, personalized support team focused on the needs of each
family throughout the Zolgensma treatment journey. This includes
answering questions about Zolgensma, verifying reimbursement
assistance, and coordinating financial assistance programs for
eligible patients. For more information, caregivers and healthcare
professionals can call 1-855-441-GENE (1-855-441-4363).
Outside of the US, Zolgensma has PRIME (PRIority MEdicines)
designation in Europe and is being
reviewed under Accelerated Assessment Procedure, and also has
accelerated Sakigake designation in Japan. In the interim, AveXis has arranged to
make the product available for international markets, subject to
local laws and regulations, as a part of its paid Managed Access
Program via a collaboration with Durbin, a third-party provider.
International inquiries regarding availability of Zolgensma outside
of the US may be made by contacting Durbin
at AveXisMAP@DurbinGlobal.com or +44-20-8869-6506.
About SMA
SMA is caused by a mutated or missing survival motor neuron 1
gene (SMN1) that prevents the body from making enough
survival motor neuron protein (SMN), ultimately leading to the loss
of motor neurons, muscle weakness, and paralysis seen in SMA. SMA
is traditionally divided into sub-types (SMA types 1, 2, 3, and 4)
based on disease onset and severity, which typically correlate to
levels of SMN protein. The most severe form of SMA is type 1,
which, without treatment, results in death or the need for
permanent breathing support by 2 years old for most patients.
Early diagnosis is key to prolonging the lifespan of infants
with SMA as the earlier the child is treated, the better the
outcomes. Newborn screening (NBS) is a program in the United States that tests newborns for a
specific set of life-threatening genetic disorders for which there
are currently treatments available, and six states now routinely
screen newborn babies for SMA.
About Zolgensma
Gene therapy uses a viral vector to deliver a missing or mutated
gene to a patient — in this case, the SMN gene — and this
new gene begins producing the missing protein. Zolgensma is a gene
therapy that targets the root cause of SMA by delivering a fully
functional SMN gene into target motor neuron cells.
Zolgensma is designed to halt disease progression by producing
sufficient and sustained levels of SMN protein required to improve
motor neuron function in a manner that has rapid onset of effect.
The therapy is a one-time intravenous infusion.
About the Muscular Dystrophy Association
MDA is committed to transforming the lives of people affected by
muscular dystrophy, ALS, and related neuromuscular diseases. We do
this through innovations in science and innovations in care. As the
largest source of funding for neuromuscular disease research
outside of the federal government, MDA has committed more than
$1 billion since our inception to
accelerate the discovery of therapies and cures. Research we have
supported is directly linked to life-changing therapies across
multiple neuromuscular diseases. MDA's MOVR is the first and only
data hub that aggregates clinical, genetic, and patient-reported
data for multiple neuromuscular diseases to improve health outcomes
and accelerate drug development. MDA supports the largest network
of multidisciplinary clinics providing best-in-class care at more
than 150 of the nation's top medical institutions. Our Resource
Center serves the community with one-on-one specialized support,
and we offer educational conferences, events, and materials for
families and healthcare providers. Each year thousands of children
and young adults learn vital life skills and gain independence at
summer camp and through recreational programs, at no cost to
families. For more information visit mda.org.
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SOURCE Muscular Dystrophy Association