– Dysport is the first and only FDA-approved
botulinum toxin for treatment of both pediatric upper and lower
limb spasticity1 –
– Pivotal Phase 3 study demonstrated Dysport
improved spasticity symptoms in children aged two to 17
experiencing upper limb spasticity, as measured by the primary
efficacy endpoint of Modified Ashworth Scale at elbow or wrist
flexors at Week 61 –
Ipsen Biopharmaceuticals, an affiliate of Ipsen (Euronext: IPN;
ADR: IPSEY), announced today that the United States Food and Drug
Administration (FDA) has expanded the use of Dysport®
(abobotulinumtoxinA) for injection to include the treatment of
upper limb spasticity in children two years of age and older,
excluding spasticity caused by cerebral palsy (CP). This approval
makes Dysport the first botulinum toxin approved by the FDA for
both pediatric spasticity indications, following the previous
approval to treat children with lower limb spasticity aged two and
older received in July 2016.1
“For physicians, it is reassuring to have a botulinum toxin
treatment in Dysport which demonstrated sustained symptom relief
for spasticity, which can be physically challenging for children,”
said Ann Tilton, MD, study investigator and Professor of Clinical
Neurology at the Louisiana State University Health Sciences Center
New Orleans. “This FDA decision for Dysport means we now have an
approved therapy to offer children and adolescents seeking
improvements in mobility in both upper and lower limbs.”
The approval is based on a Phase 3 study with children aged two
to 17 years old being treated for upper limb spasticity.1 Due to
Orphan Drug Exclusivity, this approval excludes use in children
with upper limb spasticity caused by CP. Dysport demonstrated
statistically significant improvements from baseline at Week 6 with
doses of 8 Units/kg and 16 Units/kg vs. 2 Units/kg, as measured by
the Modified Ashworth Scale (MAS) in the elbow or wrist flexors.1
Dysport demonstrated a reduction in spasticity symptoms through 12
weeks for most children for both upper and lower limbs.1 In the
upper limb study, a majority of patients were retreated between
16-28 weeks; however, some patients had a longer duration of
response (i.e., 34 weeks or more).1 The most frequent adverse
reactions observed were upper respiratory tract infection and
pharyngitis.1
Dysport and all botulinum toxin products have a Boxed Warning,
which states that the effects of the botulinum toxin may spread
from the area of injection to other areas of the body, causing
symptoms similar to those of botulism.1 Those symptoms include
swallowing and breathing difficulties that can be
life-threatening.1 Dysport is contraindicated in patients with
known hypersensitivity to any botulinum toxin preparation or to any
of the components; or in the presence of infection at the proposed
injection site(s); or in patients known to be allergic to cow’s
milk protein.1 The potency Units of Dysport are specific to the
preparation and assay method utilized.1 They are not
interchangeable with other preparations of botulinum toxin
products.1 Please see below for additional Important Safety
Information.
“This approval is a testament to Ipsen’s legacy in neurotoxin
research and continued commitment to advancing patient care,” said
Kimberly Baldwin, Vice President, Franchise Head, Neuroscience
Business Unit, Ipsen. “We believe the data for both pediatric upper
and lower limb spasticity underscore the role of Dysport as an
important treatment option for patients seeking long-lasting
spasticity symptom relief.”
Spasticity affects more than an estimated 12 million people
worldwide and is a condition in which there is an abnormal increase
in muscle tone or stiffness, which may interfere with movement and
particularly impacts growing children.2,3 Treatment with injectable
medications, including Dysport, have shown to be effective in
relieving the symptoms associated with spasticity in the arms and
legs among children.2
About Pediatric Spasticity
Spasticity is a condition in which there is an abnormal increase
in muscle tone or stiffness in one or more muscles, which might
interfere with movement.3
Spasticity affects the muscles and joints of the extremities,
and particularly impacts growing children.2 Spasticity is usually
caused by damage to nerve pathways in the brain or spinal cord that
control muscle movement, and may occur in association with CP,
spinal cord injury, multiple sclerosis, stroke, and brain or head
trauma.2,3
Symptoms of spasticity may include increased muscle tone, rapid
muscle contractions, exaggerated deep tendon reflexes, and/or
muscle spasms.2,3 The degree of spasticity can vary from mild
muscle stiffness to severe, painful, and uncontrollable muscle
spasms.3
Spasticity in children is a condition that causes muscle spasms
and increased muscle stiffness in either the upper and/or lower
limbs including the elbow, wrist, finger and calf muscles.1 When
muscle stiffness in the calf is intensified, it prohibits the ankle
from flexing as needed and causes the foot to be pointed down and
in.1,4
About the Phase 3 Pivotal Study
Dysport was evaluated in a Phase 3, randomized, double-blind,
low-dose controlled, multicenter study that included a total of 210
children treated, aged two to 17 years, for upper limb spasticity.1
Patients with a MAS of grade 2 or greater at the primary targeted
muscle groups (PTMG) were enrolled and received doses of Dysport at
8 Units/kg (n=70), 16 Units/kg (n=70) or 2 Units/kg (n=70) injected
into the PTMG (elbow flexors: brachialis and brachioradialis; wrist
flexors: flexor carpi radialis, and flexor carpi ulnaris).1 After
the initial treatment, up to three further treatments of Dysport
could be administered at planned doses of either 8 Units/kg or 16
Units/kg, or titrated up or down according to investigator
judgement.1
Dysport showed statistically significant improvements from
baseline in MAS in the PTMG at Week 6, the primary endpoint, with
doses of 8 Units/kg and 16 Units/kg compared to low dose Dysport (2
Units/kg) (-2.0, -2.3 and -1.6, respectively).1 A total of 208
patients were included in this assessment as part of the modified
intent to treat (mITT) population.1 Higher dose Dysport received a
+2.0 Physician Global Assessment (PGA) score, though there was no
statistically significant difference in mean PGA (2.0, 2.0 and 1.8,
respectively) or mean Goal Attainment Scale (GAS) (52.6, 52.6 and
52.1, respectively).1
In the upper limb study, a majority of patients were retreated
between 16-28 weeks; however, some patients had a longer duration
of response (i.e., 34 weeks or more).1
Adverse reactions (≥3%, and reported more frequently than the
control group) in pediatric patients with upper limb spasticity for
Dysport, respectively, were: upper respiratory tract infection,
pharyngitis, nausea, muscular weakness, headache, and
epilepsy.1
A pediatric assessment for Dysport demonstrates that Dysport is
safe and effective in another pediatric population. However,
Dysport is not approved for such patient population due to
marketing exclusivity for another botulinum toxin.1
About Dysport® (abobotulinumtoxinA) for
Injection
Dysport is an injectable form of botulinum toxin type A
(BoNT-A), which is isolated and purified from Clostridium bacteria
producing BoNT-A.1 It is supplied as a lyophilized powder.1 Dysport
has approved indications in the United States for the treatment of
adults with cervical dystonia (CD) and for the treatment of
spasticity in adult patients.1 Dysport is also the first and only
FDA-approved botulinum toxin for the treatment of upper and lower
limb spasticity in children two years of age or older.1
INDICATIONS AND IMPORTANT SAFETY INFORMATION
INDICATIONS
Dysport® (abobotulinumtoxinA) for injection is indicated
for the treatment of:
- Lower and upper limb spasticity in adults
- Lower limb spasticity in pediatric patients 2 years of age and
older
- Upper limb spasticity in pediatric patients 2 years of age and
older, excluding spasticity caused by cerebral palsy
- Cervical dystonia in adults
IMPORTANT SAFETY INFORMATION
Warning: Distant Spread of Toxin
Effect
Postmarketing reports indicate that the
effects of Dysport and all botulinum toxin products may spread from
the area of injection to produce symptoms consistent with botulinum
toxin effects. These may include asthenia, generalized muscle
weakness, diplopia, blurred vision, ptosis, dysphagia, dysphonia,
dysarthria, urinary incontinence, and breathing difficulties. These
symptoms have been reported hours to weeks after injection.
Swallowing and breathing difficulties can be life threatening and
there have been reports of death. The risk of symptoms is probably
greatest in children treated for spasticity, but symptoms can also
occur in adults treated for spasticity and other conditions,
particularly in those patients who have underlying conditions that
would predispose them to these symptoms. In unapproved uses and in
approved indications, cases of spread of effect have been reported
at doses comparable to or lower than the maximum recommended total
dose.
Contraindications
Dysport is contraindicated in patients with known
hypersensitivity to any botulinum toxin products, cow’s milk
protein, components in the formulation or infection at the
injection site(s). Serious hypersensitivity reactions including
anaphylaxis, serum sickness, urticaria, soft tissue edema, and
dyspnea have been reported. If such a reaction occurs, discontinue
Dysport and institute appropriate medical therapy immediately.
Warnings and Precautions
Lack of Interchangeability Between Botulinum Toxin
Products
The potency Units of Dysport are specific to the preparation and
assay method utilized. They are not interchangeable with other
preparations of botulinum toxin products, and, therefore, units of
biological activity of Dysport cannot be compared to or converted
into units of any other botulinum toxin products assessed with any
other specific assay method.
Dysphagia and Breathing Difficulties
Treatment with Dysport and other botulinum toxin products can
result in swallowing or breathing difficulties. Patients with
pre-existing swallowing or breathing difficulties may be more
susceptible to these complications. In most cases, this is a
consequence of weakening of muscles in the area of injection that
are involved in breathing or swallowing. When distant side effects
occur, additional respiratory muscles may be involved.
Deaths as a complication of severe dysphagia have been reported
after treatment with botulinum toxin. Dysphagia may persist for
several weeks, and require use of a feeding tube to maintain
adequate nutrition and hydration. Aspiration may result from severe
dysphagia and is a particular risk when treating patients in whom
swallowing or respiratory function is already compromised. Patients
treated with botulinum toxin may require immediate medical
attention should they develop problems with swallowing, speech, or
respiratory disorders. These reactions can occur within hours to
weeks after injection with botulinum toxin.
Pre-existing Neuromuscular Disorders
Individuals with peripheral motor neuropathic diseases,
amyotrophic lateral sclerosis, or neuromuscular junction disorders
(e.g., myasthenia gravis or Lambert-Eaton syndrome) should be
monitored particularly closely when given botulinum toxin. Patients
with neuromuscular disorders may be at increased risk of clinically
significant effects including severe dysphagia and respiratory
compromise from typical doses of Dysport.
Human Albumin and Transmission of Viral Diseases
This product contains albumin, a derivative of human blood.
Based on effective donor screening and product manufacturing
processes, it carries an extremely remote risk for transmission of
viral diseases and variant Creutzfeldt-Jakob disease (vCJD). There
is a theoretical risk for transmission of Creutzfeldt-Jakob disease
(CJD), but if that risk actually exists, the risk of transmission
would also be considered extremely remote. No cases of transmission
of viral diseases, CJD, or vCJD have ever been identified for
licensed albumin or albumin contained in other licensed
products.
Intradermal Immune Reaction
The possibility of an immune reaction when injected
intradermally is unknown. The safety of Dysport for the treatment
of hyperhidrosis has not been established. Dysport is approved only
for intramuscular injection.
Most Common Adverse Reactions
Adults with lower limb spasticity (≥5%): falls, muscular
weakness, and pain in extremity and with upper limb
spasticity (≥4%): muscular weakness.
Pediatric patients with lower limb spasticity (≥10%):
nasopharyngitis, cough and pyrexia and with upper limb
spasticity (≥10%): upper respiratory tract infection and
pharyngitis.
Adults with cervical dystonia (≥5%): muscular weakness,
dysphagia, dry mouth, injection site discomfort, fatigue, headache,
musculoskeletal pain, dysphonia, injection site pain, and eye
disorders.
Drug Interactions
Co-administration of Dysport and aminoglycosides or other agents
interfering with neuromuscular transmission (e.g., curare-like
agents), or muscle relaxants, should be observed closely because
the effect of botulinum toxin may be potentiated. Use of
anticholinergic drugs after administration of Dysport may
potentiate systemic anticholinergic effects, such as blurred
vision. The effect of administering different botulinum neurotoxins
at the same time or within several months of each other is unknown.
Excessive weakness may be exacerbated by another administration of
botulinum toxin prior to the resolution of the effects of a
previously administered botulinum toxin. Excessive weakness may
also be exaggerated by administration of a muscle relaxant before
or after administration of Dysport.
Special Populations
Use in Pregnancy
There are no adequate and well-controlled studies in pregnant
women. Dysport should be used during pregnancy only if the
potential benefit justifies the potential risk to the fetus. Based
on animal data, Dysport may cause fetal harm.
Pediatric Use
The safety and effectiveness of Dysport injected into proximal
muscles of the lower limb for the treatment of spasticity in
pediatric patients has not been established. Based on animal data
Dysport may cause atrophy of injected and adjacent muscles;
decreased bone growth, length, and mineral content; delayed sexual
maturation; and decreased fertility.
Geriatric Use
In general, elderly patients should be observed to evaluate
their tolerability of Dysport, due to the greater frequency of
concomitant disease and other drug therapy. Subjects aged 65 years
and over who were treated with Dysport for lower limb spasticity
reported a greater percentage of fall and asthenia as compared to
those younger (10% vs. 6% and 4% vs. 2%, respectively).
To report SUSPECTED ADVERSE REACTIONS or product complaints,
contact Ipsen at 1-855-463-5127. You may also report SUSPECTED
ADVERSE REACTIONS to the FDA at 1-800-FDA-1088 or
www.fda.gov/medwatch.
Please see full Prescribing Information, including Boxed
Warning and Medication Guide.
About Ipsen in North America
Ipsen (Euronext: IPN; ADR: IPSEY) is a global biopharmaceutical
company focused on innovation and specialty care. The company
develops and commercializes innovative medicines in three key
therapeutic areas – Oncology, Neuroscience and Rare Diseases. At
Ipsen, we focus our resources, investments and energy on
discovering, developing and commercializing new therapeutic options
to provide hope for patients whose lives are challenged by
difficult-to-treat diseases. Ipsen’s North American operations are
located in Cambridge, Massachusetts, one of the company’s three
global hubs. Based in the heart of Kendall Square, our fully
integrated biopharmaceutical business includes Commercial, Research
& Development, Manufacturing, and Global External Innovation
and Partnering. Combined with our Canadian headquarters in
Mississauga, Ontario, and other locations, Ipsen employs
approximately 600 people in North America. For more information
please visit www.ipsenus.com or www.ipsen.ca. Connect with us on
Twitter and LinkedIn.
About Ipsen
Ipsen is a global specialty-driven biopharmaceutical group
focused on innovation and specialty care. The group develops and
commercializes innovative medicines in three key therapeutic areas
– Oncology, Neuroscience and Rare Diseases. Its commitment to
Oncology is exemplified through its growing portfolio of key
therapies for prostate cancer, neuroendocrine tumors, renal cell
carcinoma and pancreatic cancer. Ipsen also has a well-established
Consumer Healthcare business. With total sales over €2.2 billion in
2018, Ipsen sells more than 20 drugs in over 115 countries, with a
direct commercial presence in more than 30 countries. Ipsen’s
R&D is focused on its innovative and differentiated
technological platforms located in the heart of the leading
biotechnological and life sciences hubs (Paris-Saclay, France;
Oxford, UK; Cambridge, US). The Group has about 5,700 employees
worldwide. Ipsen is listed in Paris (Euronext: IPN) and in the
United States through a Sponsored Level I American Depositary
Receipt program (ADR: IPSEY). For more information on Ipsen, visit
www.ipsen.com.
Forward-Looking Statement
The forward-looking statements, objectives and targets contained
herein are based on the Group’s management strategy, current views
and assumptions. Such statements involve known and unknown risks
and uncertainties that may cause actual results, performance or
events to differ materially from those anticipated herein. All of
the above risks could affect the Group’s future ability to achieve
its financial targets, which were set assuming reasonable
macroeconomic conditions based on the information available today.
Use of the words "believes", "anticipates" and "expects" and
similar expressions are intended to identify forward-looking
statements, including the Group’s expectations regarding future
events, including regulatory filings and determinations, and the
outcome of this study or other studies. Moreover, the targets
described in this document were prepared without taking into
account external growth assumptions and potential future
acquisitions, which may alter these parameters. These objectives
are based on data and assumptions regarded as reasonable by the
Group. These targets depend on conditions or facts likely to happen
in the future, and not exclusively on historical data. Actual
results may depart significantly from these targets given the
occurrence of certain risks and uncertainties, notably the fact
that a promising product in early development phase or clinical
trial may end up never being launched on the market or reaching its
commercial targets, notably for regulatory or competition reasons.
The Group must face or might face competition from generic products
that might translate into a loss of market share. Furthermore, the
Research and Development process involves several stages each of
which involves the substantial risk that the Group may fail to
achieve its objectives and be forced to abandon its efforts with
regards to a product in which it has invested significant sums.
Therefore, the Group cannot be certain that favorable results
obtained during pre-clinical trials will be confirmed subsequently
during clinical trials, or that the results of clinical trials will
be sufficient to demonstrate the safe and effective nature of the
product concerned. There can be no guarantees a product will
receive the necessary regulatory approvals or that the product will
prove to be commercially successful. If underlying assumptions
prove inaccurate or risks or uncertainties materialize, actual
results may differ materially from those set forth in the
forward-looking statements. Other risks and uncertainties include
but are not limited to, general industry conditions and
competition; general economic factors, including interest rate and
currency exchange rate fluctuations; the impact of pharmaceutical
industry regulation and health care legislation; global trends
toward health care cost containment; technological advances, new
products and patents attained by competitors; challenges inherent
in new product development, including obtaining regulatory
approval; the Group's ability to accurately predict future market
conditions; manufacturing difficulties or delays; financial
instability of international economies and sovereign risk;
dependence on the effectiveness of the Group’s patents and other
protections for innovative products; and the exposure to
litigation, including patent litigation, and/or regulatory actions.
The Group also depends on third parties to develop and market some
of its products which could potentially generate substantial
royalties; these partners could behave in such ways which could
cause damage to the Group’s activities and financial results. The
Group cannot be certain that its partners will fulfil their
obligations. It might be unable to obtain any benefit from those
agreements. A default by any of the Group’s partners could generate
lower revenues than expected. Such situations could have a negative
impact on the Group’s business, financial position or performance.
The Group expressly disclaims any obligation or undertaking to
update or revise any forward-looking statements, targets or
estimates contained in this press release to reflect any change in
events, conditions, assumptions or circumstances on which any such
statements are based, unless so required by applicable law. The
Group’s business is subject to the risk factors outlined in its
registration documents filed with the French Autorité des Marchés
Financiers. The risks and uncertainties set out are not exhaustive
and the reader is advised to refer to the Group’s 2018 Registration
Document available on its website (www.ipsen.com).
DYSPORT is a registered trademark of Ipsen
Biopharm Limited ©2019 Ipsen Biopharmaceuticals, Inc. September
2019 DYS-US-004024
1 Dysport (abobotulinumtoxinA) [Prescribing Information].
Cambridge, MA: Ipsen Biopharmaceuticals, Inc; September 2019. 2
American Association of Neurological Surgeons. Spasticity page.
http://www.aans.org/Patients/Neurosurgical-Conditions-and-Treatments/Spasticity.
Accessed September 16, 2019. 3 National Institute of Neurological
Disorders and Stroke. Spasticity Information Page.
https://www.ninds.nih.gov/Disorders/All-Disorders/Spasticity-Information-Page.
Accessed September 16, 2019. 4 Delgado MR, Tilton A, Russman B, et
al. (2016). AbobotulinumtoxinA for Equinus Foot Deformity in
Cerebral Palsy: A Randomized Controlled Trial. Pediatrics.
2016;137(2);1-9.
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version on businesswire.com: https://www.businesswire.com/news/home/20190926005480/en/
For further information: Maryann Quinn Director, Product
Communications Tel: +1-857-529-1151 E-mail:
maryann.quinn@ipsen.com
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