FDA Accepts sBLA for BOTOX® (onabotulinumtoxinA) to Treat Neurogenic Detrusor Overactivity in Children Aged 5–17

June 24, 2020 /BioValleyBIOON/ -- AbbVie recently announced that the U.S. Food and Drug Administration (FDA) has accepted a supplemental Biologics License Application (sBLA) submitted to expand the prescribing information for Botox (Bao Tuoshi, generic name: onabotulinumtoxinA, botulinum toxin type A) for pediatric patients (aged 5–17 years) who have an inadequate response to, are intolerant of, or are unwilling to continue anticholinergic therapy for any reason, for the treatment of symptoms and signs of detrusor (bladder muscle) overactivity associated with an underlying neurological condition (such as spina bifida or spinal cord injury).

BOTOX® is the first and only neurotoxin approved for the treatment of urinary leakage (incontinence) in adult patients due to overactive bladder caused by neurological conditions. It is specifically indicated for adult patients who continue to experience urinary leakage or cannot tolerate the side effects after trying anticholinergic medications. BOTOX® works by selectively blocking the release of the neurotransmitter acetylcholine at the neuromuscular junction, thereby preventing nerve impulses from being transmitted to muscles (in this indication, the bladder muscle), temporarily reducing muscle contractions.

This sBLA aims to expand the indicated population for BOTOX® to include children aged 5 to 17 years. The sBLA is based on data from a randomized, double-blind Phase III study evaluating the safety and efficacy of Botox in more than 100 pediatric patients with neurogenic detrusor overactivity, as well as from a long-term extension study. The sBLA will be reviewed under the standard 10-month review cycle, with a Prescription Drug User Fee Act (PDUFA) target date in the first quarter of 2021.

Neurogenic Detrusor Overactivity (Image source: epainassist.com)

“Dr. Mitchell F. Brin, Chief Scientific Officer and Senior Vice President of Botox and Neurotoxins at AbbVie, stated: “FDAThe acceptance of this application underscores our ongoing commitment to pursuing the full potential of BOTOX® to serve patients with a variety of conditions and clinical needs. Currently, treatment options for children with neurogenic detrusor overactivity (NDO) who have failed anticholinergic therapy and prior to undergoing surgery are very limited. If approved, BOTOX® would be the first neurotoxin therapy approved for the treatment of pediatric patients with NDO who have an inadequate response to anticholinergics.

Paul F. Austin, M.D., Chief of Pediatric Urology at Texas Children’s Hospital and Professor of Urology at Baylor College of Medicine, stated, “Over time, many pediatric patients with underlying neurological conditions develop bladder and kidney damage, making treatment essential. Current treatment regimens typically include anticholinergic medications, which require careful consideration for long-term use in addition to surgical options. BOTOX® has demonstrated promising clinical outcomes in pediatric patients with neurogenic detrusor overactivity, addressing an unmet and ongoing medical need among children and adolescents.”

Neurogenic detrusor overactivity stems from ineffective communication between the spinal cord and the bladder. As a result, the bladder muscle (detrusor) contracts involuntarily, increasing intravesical pressure and reducing bladder capacity, which leads to frequent and involuntary urine leakage in patients. If not adequately treated with clean intermittent catheterization and anticholinergic medications, this condition may require augmentation cystoplasty (an extensive surgical procedure that enlarges the bladder using a segment of the patient’s intestine) to prevent renal damage.

Pediatric neurogenic detrusor overactivity has many causes, such as transverse myelitis, spinal cord injury, and spina bifida. Among these, spina bifida is the most common cause, affecting 1,500 to 2,000 out of more than 4 million infants annually in the United States. Over 90% of patients with spina bifida exhibit urinary symptoms.

Botox (Botox), developed by Allergan (acquired by AbbVie), has botulinum toxin type A as its main active ingredient. It is a neurotransmission blocker used to treat overactive muscles. Botox was first approved in 1989 for the treatment of blepharospasm and strabismus, and later approved in 2000 for cervical dystonia. Its applications have since expanded into the cosmetic field, including wrinkle reduction, facial slimming, and the elimination of glabellar lines and crow's feet. In recent years, Botox has also been approved for multiple indications, such as upper limb spasticity, chronic migraine, neurogenic urinary incontinence, overactive bladder, spasticity, and severe axillary hyperhidrosis (excessive underarm sweating).

In the United States, to date, Botox has beenFDAApproved for 11 therapeutic indications. Over the past 30 years, more than 100 million vials of Botox® and Botox® Cosmetic (onabotulinumtoxinA) have been sold worldwide, with over 3,700 articles published in scientific and medical journals. Botox® neurotoxin is one of the most extensively studied drugs globally. (Bioon.com)

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