- Making it the first and only botulinum toxin
in the UK to be approved for the treatment of pediatric spasticity
in both upper and lower limbs
- The first botulinum toxin approval in
pediatric upper limb spasticity in Europe in over a decade
Regulatory News:
Ipsen (Euronext: IPN; ADR: IPSEY) today announced that the UK’s
Medicines and Healthcare Products Regulatory Agency (MHRA) has
granted a licence update for Dysport® for the symptomatic treatment
of focal spasticity of upper limbs in pediatric cerebral palsy
patients, two years of age and older.
Spasticity in children is most commonly associated with cerebral
palsy (CP).1,2 Approximately 17 million people worldwide are
affected by cerebral palsy, with an estimated 1 in 400 babies born
with cerebral palsy in the UK, 75-91% of whom will have a specific
type known as spastic cerebral palsy.1,2 For these children,
spasticity affects muscle tone, movement, and motor skills,
hindering their ability to move in a coordinated and purposeful
way, which can consequently impact on their ability to participate
in everyday activities.3
“Therapeutic options such as botulinum toxin type A are an
important part of the multidisciplinary approach for treating
spasticity,” said Alison Smith, Consultant Pediatric
Neuro-physiotherapist, NPP Neuro Group, UK. “They work by
interrupting the muscle contraction and thereby reducing stiffness
related to spasticity with the aim of helping children with
cerebral palsy to not only improve physical functioning but also
achieve their goals which can improve their mental and emotional
wellbeing. Having a therapeutic option approved for both upper and
lower limb indications creates a real benefit for the patient as it
allows a holistic treatment approach for any patients with
multi-focal spasticity.”
This approval was based on the Phase III study demonstrating
that Dysport® reduced spasticity symptoms in children aged two
years and older being treated for upper limb spasticity due to
cerebral palsy, as measured by the Modified Ashworth Scale (MAS),
which is the standard scale for assessing muscle resistance
associated with spasticity.4 The safety profile was consistent with
that seen in the approved indications for pediatric cerebral palsy
lower limb spasticity after repeated injections and no new safety
concerns were identified.5
Asad Mohsin Ali, UK & Ireland General Manager, Ipsen said
“Today’s approval is an important advancement for children in the
UK living with cerebral palsy, who can now benefit from
long-lasting symptom relief between their botulinum toxin A
injections. As a father myself, I am proud that Ipsen is the first
company to have obtained this approval that may help children live
as normal a life as possible.”
Effective treatment of spasticity requires a highly specialized,
multidisciplinary approach including physiotherapy and occupational
therapy to reduce overactivity and the risk of permanent muscle
shortening, thus promoting functional activity and helping to allow
the child to participate in their daily activities.6
About Pediatric Cerebral Palsy Spasticity
Spasticity is abnormal and involuntary muscle stiffness, or
overactivity (contractions) in a group of muscles7, which causes
them to have increased tone, leading to stiffness or tightness.8
Cerebral palsy (CP) is the leading cause of childhood disability
affecting function and development, and the most frequent cause of
spasticity in children.3 Approximately 17 million people worldwide
are affected by cerebral palsy, with an estimated 1 in 400 babies
born in the UK have a type of CP, approximately 90% of whom will
develop spastic cerebral palsy.1,2
Upper limb spasticity in children is a condition that causes
muscle spasms in the elbow, wrist, and finger muscles.9 Lower limb
spasticity is a condition that causes increased muscle stiffness in
the calf, which, can prevent the ankle from flexing as needed and
causes the foot to be pointed down and in.10 Upper limb is the most
common form of spasticity and is a significant source of disability
particularly in children where impaired muscle growth can lead to
abnormal posturing and deformities causing pain and difficulties
performing daily tasks such as washing.11,12
About the Phase III Pivotal Study
Dysport® was evaluated in a Phase III, 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.13 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 or wrist flexors: flexor carpi radialis, and
flexor carpi ulnaris).13 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.13 Primary endpoint was mean
change in MAS score from baseline to Treatment 1 at week 6 in
Primary Targeted Muscle Group (elbow flexors or wrist flexors);
secondary endpoints were mean Physician Global Assessment (PGA)
score and Goal Attainment Scale (GAS) score at week 6. Spasticity
improvements were also assessed using the Tardieu scale as a
tertiary endpoint. Also included were safety assessments.13
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).14 A total of 208
patients were included in this assessment as part of the modified
intent to treat (mITT) population.14 Dysport® (16 Units/kg)
received a mean +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) between groups.14 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).14 The safety profile was
consistent with that seen in the approved indications for pediatric
cerebral palsy lower limb spasticity after repeated injections and
no new safety concerns were identified.5
About Dysport®
Dysport® is an injectable form of a botulinum neurotoxin type A
product, which is a substance derived from Clostridium bacteria
producing botulinum toxin type A (BoNT-A) that inhibits the
effective transmission of nerve impulses and thereby reduces
muscular contractions.15 It is supplied as a lyophilized powder. As
of 31 December 2018, Dysport® had marketing authorization in more
than 85 countries for therapeutic treatment indications and more
than 30 years of clinical experience. Dysport® was first approved
in the U.K. in 1990 for the treatment of blepharospasm and
hemifacial spasm.16
Dysport® is indicated for symptomatic treatment of focal
spasticity of upper limbs in adults, lower limbs in adults
affecting the ankle joint due to stroke or traumatic brain injury
(TBI) and dynamic equinus foot deformity in ambulant pediatric
cerebral palsy patients, two years of age or older. Dysport® is
also indicated in adults for symptomatic treatment of spasmodic
torticollis, blepharospasm, hemifacial spasm and severe primary
hyperhidrosis of the axillae, which does not respond to topical
treatment with antiperspirants or antihidrotics.
Dysport® should only be administered by appropriately trained
physicians. For the treatment of focal spasticity, Dysport® can
also be administered by healthcare professionals having received
appropriate training and qualification in accordance with national
guidelines (e.g. Royal College of Physicians).2
Ipsen co-developed Dysport® in partnership with the UK
Government bodies, specifically the Center for Applied Microbiology
and Research14 and provides continued value through a quarterly
royalty to Public Health England which totaled more than £30m in
2018.17
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
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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. 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
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regards to a product in which it has invested significant sums.
Therefore, the Group cannot be certain that favorable results
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References
- Shamsoddini, Alireza et al. Management of spasticity in
children with cerebral palsy. Iranian journal of pediatrics 2014;
24(4): 345-51.
- Cerebral Palsy Foundation. Key Facts. Available at:
www.yourcpf.org/statistics. Accessed November 2019.
- Cerebral Palsy Sport. Key facts and statistics. Available at:
http://www.cpsport.org/resources/cerebral-palsy-key-facts-and-statistics/
Accessed November 2019.
- Craven, B., Morris, A. Modified Ashworth scale reliability for
measurement of lower extremity spasticity among patients with SCI.
Spinal Cord 2010; 48: 207–213.
- Ipsen. Data on File.
- Hoare, J Child Neurol. 2014; 29(8):1066-76.
- The Hillingdon hospitals NHS Foundation Trust. Patient
information leaflet. Spasticity and its management. Last accessed:
November 2019.
- Physical effects of stroke. Stroke Association
www.stroke.org.uk. Last accessed: November 2019.
- Lynn Bar-On et al. Spasticity and its contribution to
hypertonia in cerebral palsy. Biomed Res Int. 2015; 2015:
317047.
- Pirazzini, M., Rossetto, O., Eleopra, R. & Montecucco, C.
Pharmacol. Rev. 200–235 (2017). doi:10.1124/pr.116.012658
- Santos CA, Franco de Moura RC, Lazzari RD, Dumont AJ, Braun LA,
Oliveira CS. J Phys Ther Sci 2015; 27(5): 1617-20.
- Colver A, Fairhurst C, Pharoah PO. Cerebral palsy. Lancet 2014;
383(9924): 1240-9.
- SmPC Dysport 500 Units. 175.1_DYS500_UK.
- Erbguth FJ. J Nerual Transm 2007;115: 559-65.
- Jitpimolmard S, et al. J Neurol Neurosurg Psychiatry
1998;64(6):751-757.
- Ipsen. Neuroscience. Available
from:https://www.ipsen.com/our-science/neuroscience/. Accessed
November 2019.
- Ipsen. Data on File. DYS-UK-003296. May 2019.
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For further information: Ipsen Christian Marcoux Senior
Vice President, Global Communications +33 (0) 1 58 33 67 94
christian.marcoux@ipsen.com
Kelly Blaney Vice President, Global Communications +44 (0) 7903
402275 kelly.blaney@ipsen.com
Eugenia Litz Vice President, Investor Relations +44 (0) 1753
627721 eugenia.litz@ipsen.com
Myriam Koutchinsky Investor Relations Manager +33 (0) 158 33 51
04 myriam.koutchinsky@ipsen.com
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