FDA Oks Botox As a Biologic for Pediatric Urology Patients

June 25, 2020 - Allergan, an AbbVie company, recently announced that the FDA accepted its supplemental biologics license application (sBLA) to expand BOTOX prescribing information for bladder (detrusor) overactivity associated with underlying neurologic conditions.

BOTOX (Onabotulinumtoxin A) is the first and only neurotoxin approved for the treatment of leakage of urine (incontinence) due to overactive bladder caused by a neurologic condition. It is intended for pediatric patients who have inadequate responses to, or are intolerant of, anticholinergic medication.

The sBLA is based on data from a randomized, double-blind Phase 3 study evaluating the safety and efficacy of BOTOX in over 100 patients with neurogenic bladder (detrusor) overactivity and long-term extension study.

"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," Mitchell F. Brin, MD, senior vice president, chief scientific officer, BOTOX & neurotoxins, AbbVie, said in the announcement. 

"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 ineffective communication between the spinal cord and bladder. Because of this, the detrusor muscle contracts, which can cause the individual to urinate frequently and unexpectedly.

BOTOX temporarily reduces muscle contractions by blocking the transmission of nerve impulses to the bladder muscle, AbbVie said. But this condition may require a surgical procedure to prevent renal damage if not efficiently managed with anticholinergic drugs.

Many children with chronic conditions experience neurogenic detrusor overactivity, including individuals with transverse myelitis, spinal cord injury, and spina bifida, which is the most common and affects 1,500 to 2,000 of the more than four million babies born in the US each year, AbbVie stated.

FDA first approved BOTOX in 1989 for two rare eye muscle disorders – blepharospasm and strabismus in adults.

Currently, BOTOX is FDA-approved for 11 therapeutic indications, including chronic migraine, multiple sclerosis (MS), cervical dystonia, spasticity, and severe underarm sweating (axillary hyperhidrosis), AbbVie said. 

Urinary incontinence is believed to have a greater social impact than any other MS symptom or complication when it comes to quality of life, according to an article. 

Botulinum toxin is a muscle-relaxing medication that’s made from bacteria called Clostridium botulinum and is extremely helpful for individuals with MS who have spasticity, stiff muscles, and sudden uncontrollable movements.

The benefits include a significant improvement in bladder control, patient satisfaction, and urodynamic parameters at up to 9-month follow-up. Repeat injections are as effective as the first injection, according to an article published in International Journal of MS Care.

"Over time, many pediatric patients with underlying neurologic conditions experience bladder and kidney damage, and treatment is critical," Paul F. Austin, MD, FAAP, chief of pediatric urology at Texas Children's Hospital and professor of urology at Baylor College of Medicine, said in the Allergan announcement. 

"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."