Ipsen Announces FDA Approval of Dysport® (abobotulinumtoxinA) for the Treatment of Upper Limb Spasticity in Children, Excluding Cerebral Palsy

– 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

CAMBRIDGE, MA, USA I September 26, 2019 I 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

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.


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.

SOURCE: Ipsen

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