FDA Expands Use of Botox to include Rare Pediatric Neurologic Condition

Rare Daily Staff

The U.S. Food and Drug Administration granted expanded approval to Allergan’s Botox for the treatment of detrusor overactivity associated with a neurologic condition in pediatric patients 5 years of age and older who have an inadequate response to or are intolerant of anticholinergic medication.

It is the twelfth approved indication for Botox and the first neurotoxin approved for use in treating neurogenic detrusor overactivity.

Neurogenic detrusor overactivity occurs when the spinal cord and bladder are not able to communicate effectively, which can occur with neurologic conditions such as spina bifida and spinal cord injuries. As a result, the bladder muscle involuntarily contracts, increasing the pressure in the bladder and reducing the bladder capacity, which can cause the individual to leak urine frequently and unexpectedly. Elevated bladder pressure can also lead to bladder and kidney damage over time.

There are several causes of neurogenic detrusor overactivity in children, such as transverse myelitis, spinal cord injury, and spina bifida, the latter of 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 percent of those living with spina bifida experience urinary symptoms.

The FDA approval 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. Results from the phase 3 study demonstrated that intradetrusor administration of Botox 200 Units (not to exceed 6U/kg) reduced daytime urinary incontinence episodes, the study’s primary endpoint, as well as lowered maximum bladder pressure and increased bladder capacity at week 6.

The most common adverse reaction in the studies were bacteriuria (20 percent), urinary tract infection (7 percent), leukocyturia (7 percent), and hematuria (3 percent).

“Many children with underlying neurologic conditions may experience bladder and kidney damage over time, which underscores the importance of treatment. When caring for pediatric patients with neurogenic detrusor overactivity, we strive to reduce bladder pressure and increase the bladder’s capacity,” said Paul Austin, chief of pediatric urology at Texas Children’s Hospital and professor of urology at Baylor College of Medicine. “Previously, treatment options were limited primarily to anticholinergic medicines, where long-term use needs to be considered carefully, in addition to surgery. Effectively managing neurogenic detrusor overactivity requires ongoing care, and there has been a high unmet need for alternative treatments.”

Photo: Paul Austin, chief of pediatric urology at Texas Children’s Hospital and professor of urology at Baylor College of Medicine