FDA Accepts Supplemental Biologics License Application (sBLA) for BOTOX® (onabotulinumtoxinA) for the Treatment of Pediatric Patients with Neurogenic Detrusor Overactivity

- Application Seeks to Extend Use of BOTOX® for Patients 5 to 17 Years OldNORTH

CHICAGO, IL, USA I June 22, 2020 I Allergan, an AbbVie (NYSE: ABBV) company, today announced that the U.S. Food and Drug Administration (FDA) has accepted the company's supplemental biologics license application (sBLA) to expand the BOTOX® prescribing information for the treatment of signs and symptoms of detrusor (bladder muscle) overactivity associated with an underlying neurologic condition (e.g., spina bifida, spinal cord injuries) in pediatric patients (5 -17 years of age) who have an inadequate response to, or are intolerant of, or for any reason unwilling to continue anticholinergic medication.

The sBLA is based on data from a randomized, double-blind Phase 3 study evaluating the safety and efficacy of BOTOX® in more than 100 pediatric patients with neurogenic detrusor overactivity and a long-term extension study. The Prescription Drug User Fee Act date is expected to be in the first quarter of 2021 following a standard 10-month review.

"The FDA acceptance of this application underscores our ongoing commitment to pursuing the full potential of BOTOX® to serve patients across a wide spectrum of diseases and clinical needs," said Mitchell F. Brin, M.D., Senior Vice President, Chief Scientific Officer, BOTOX® & Neurotoxins, AbbVie. "Children living with neurogenic detrusor overactivity currently have limited options when they fail anticholinergic medications and prior to surgical intervention. If approved, BOTOX® will be the first neurotoxin treatment approved for use in treating detrusor overactivity in pediatric patients with an underlying neurologic condition who are not adequately managed by anticholinergic medications."

Neurogenic detrusor overactivity results from the inability of the spinal cord and bladder to communicate effectively. As a result, the bladder muscle (detrusor) involuntarily contracts, increasing the pressure in the bladder and reducing the bladder capacity, which can cause the individual to leak urine frequently and unexpectedly. If not adequately managed with clean intermittent catheterization and anticholinergic drugs, this condition may require augmentation cystoplasty (an extensive surgical procedure in which the bladder is enlarged using a section of the patient's intestine) to prevent renal damage.

There are a number of causes of neurogenic detrusor overactivity in children, such as transverse myelitis, spinal cord injury, and spina bifida, which is the most common and affects 1,500-2,000 of the more than 4 million babies born in the United States each year.1 More than 90% of those living with spina bifida experience urinary symptoms.2,3,4,5 

"Over time, many pediatric patients with underlying neurologic conditions experience bladder and kidney damage, and treatment is critical," said Paul F. Austin, M.D., FAAP, Chief of Pediatric Urology at Texas Children's Hospital and Professor of Urology at Baylor College of Medicine. "Current treatment options often include anticholinergic medications, where long-term use needs to be considered carefully, in addition to surgery. The favorable BOTOX® clinical results for the treatment of pediatric patients with neurogenic detrusor activity are promising as we look to address unmet and ongoing needs in children and adolescents."

BOTOX® is the first and only neurotoxin approved for the treatment of leakage of urine (incontinence) due to overactive bladder caused by a neurologic condition in adults who still have leakage or cannot tolerate the side effects after trying an anticholinergic medication. BOTOX® temporarily reduces muscle contractions by blocking the transmission of nerve impulses to the muscle, in this case, the bladder muscle, by selectively preventing the release of the neurotransmitter acetylcholine at the neuromuscular junction.

About BOTOX®
BOTOX® is one of the most widely researched medications in the world, with a proven history as a therapeutic agent.6 First approved by the FDA in 1989 for two rare eye muscle disorders – blepharospasm and strabismus in adults, BOTOX® was the world's first approved botulinum toxin type A treatment. Today, BOTOX® is FDA-approved for 11 therapeutic indications, including Chronic Migraine, overactive bladder, leakage of urine (incontinence) due to overactive bladder caused by a neurologic condition, cervical dystonia, spasticity, and severe underarm sweating (axillary hyperhidrosis). Backed by strong science and continuous innovation, BOTOX® proudly embraces its past while boldly looking to the future.

BOTOX® (onabotulinumtoxinA) Important Information

Indications
BOTOX® is a prescription medicine that is injected into muscles and used:

  • To treat overactive bladder symptoms such as a strong need to urinate with leaking or wetting accidents (urge urinary incontinence), a strong need to urinate right away (urgency), and urinating often (frequency) in adults 18 years and older when another type of medicine (anticholinergic) does not work well enough or cannot be taken
  • To treat leakage of urine (incontinence) in adults 18 years and older with overactive bladder caused by a neurologic disease who still have leakage or cannot tolerate the side effects after trying an anticholinergic medication
  • To prevent headaches in adults with Chronic Migraine who have 15 or more days each month with headache lasting 4 or more hours each day in people 18 years or older
  • To treat increased muscle stiffness in elbow, wrist, finger, and thumb muscles in people 18 years and older with upper limb spasticity
  • To treat increased muscle stiffness in ankle and toe muscles in people 18 years and older with lower limb spasticity
  • To treat increased muscle stiffness in children 2 to 17 years of age with upper limb spasticity
  • To treat increased muscle stiffness in children 2 to 17 years of age with lower limb spasticity, excluding spasticity caused by cerebral palsy
  • To treat the abnormal head position and neck pain that happens with Cervical Dystonia (CD) in people 16 years and older
  • To treat certain types of eye muscle problems (Strabismus) or abnormal spasm of the eyelids (Blepharospasm) in people 12 years and older

BOTOX® is also injected into the skin to treat the symptoms of severe underarm sweating (severe primary axillary hyperhidrosis) when medicines used on the skin (topical) do not work well enough in people 18 years and older.

It is not known whether BOTOX® is safe or effective to prevent headaches in patients with migraine who have 14 or fewer headache days each month (episodic migraine).

It is not known whether BOTOX® is safe or effective to treat increased stiffness in upper limb muscles other than those in the elbow, wrist, fingers, and thumb, or in lower limb muscles other than those in the ankle and toes in people 18 years and older. BOTOX® has not been shown to help people perform task-specific functions with their upper limbs or increase movement in joints that are permanently fixed in position by stiff muscles.

It is not known whether BOTOX® is safe or effective for other types of muscle spasms or for severe sweating anywhere other than your armpits.

Please see BOTOX® full Product Information, including Boxed Warning and Medication Guide.

About AbbVie

AbbVie's mission is to discover and deliver innovative medicines that solve serious health issues today and address the medical challenges of tomorrow. We strive to have a remarkable impact on people's lives across several key therapeutic areas: immunology, oncology, neuroscience, eye care, virology, women's health and gastroenterology, in addition to products and services across its Allergan Aesthetics portfolio. For more information about AbbVie, please visit us at www.abbvie.com. Follow @abbvie on Twitter, Facebook, Instagram, YouTube and LinkedIn.

References:

  1. National Institute of Neurological Disorders and Stroke. Spina Bifida Fact Sheet. Accessed online 5/6/2020 and available from: https://www.ninds.nih.gov/disorders/patient-caregiver-education/fact-sheets/spina-bifida-fact-sheet.
  2. Hascoet J, Manunta A, Brochard C, Arnaud A, Damphousse M, Menard H, et al. Outcomes of intra-detrusor injections of botulinum toxin in patients with spina bifida: A systematic review. Neurourology and Urodynamics. 2017;36(3):557-564.
  3. Pannek J, Blok B, Castro-Diaz D, Del Popolo G, Kramer G, Radziszewski P, et. al. Guidelines on neurogenic lower urinary tract dysfunction. European Association of Urology, 2013. Available from: https://uroweb.org/wp-content/uploads/20_Neurogenic-LUTD_LR.pdf.
  4. Shin M, Besser LM, Siffel C, Kucik JE, Shaw GM, Lu C, Correa A; Congenital Anomaly Multistate Prevalence and Survival Collaborative. Prevalence of spina bifida among children and adolescents in 10 regions in the United States. Pediatrics. 2010;126(2):274-279.
  5. Ouyang L, Grosse SD, Armour BS, Waitzman NJ. Health care expenditures of children and adults with spina bifida in a privately insured U.S. population. Birth Defects Research Part A, Clinical and Molecular Teratology. 2007;79(7):552-558.
  6. Data on file, Allergan; BOTOX® Worldwide Marketing Authorization Status; BOTOX® Prescribing Information.

SOURCE: AbbVie

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