FDA Accepts sBLA for BOTOX for Neurogenic Detrusor Overactivity

Allergan, an AbbVie company, announced 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. More than 90% of those living with spina bifida experience urinary symptoms.

"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.