AbbVie recently announced that the United States Food and Drug Administration (FDA) has accepted a supplementary biological product license application (sBLA) submitted by it, which seeks to expand the prescription information of Botox (common name: onabotulinumtoxinA). For pediatric patients (5-17 years old) with insufficient response, intolerance or unwilling to continue anticholinergic drugs for any reason, treatment and an intrinsic nervous system disease (such as spina bifida, spinal cord injury) ) Related symptoms and signs of detrusor (bladder muscle) overactivity.
BOTOX is the first and only neurotoxin approved for adult patients to treat urinary leakage (incontinence) caused by overactive bladder caused by neurological diseases. The drug is specifically suitable for: after trying anticholinergic drugs There are still adult patients with urine leakage or intolerance of side effects. BOTOX selectively blocks the release of the neurotransmitter acetylcholine at the neuromuscular junction by temporarily blocking the transmission of nerve impulses to the muscle (in this indication, the bladder muscle), temporarily reducing muscle contraction.
The sBLA aims to expand the applicable population of BOTOX® for children aged 5-17. The sBLA was based on data from a randomized, double-blind phase III study and a long-term extended study evaluating the safety and effectiveness of Botox in more than 100 children with neurogenic detrusor overactivity. The sBLA will be reviewed through a standard 10-month review cycle. The target date for the prescription drug user fee method (PDUFA) is the first quarter of 2021.
Neurogenic detrusor overactivity (Image source: epainassist.com)
AbbVie Botox and Neurotoxin Chief Scientific Officer and Senior Vice President Mitchell F. Brin, M.D., said: "The FDA's acceptance of this application underscores our continued commitment to pursuing the full potential of BOTOX® to serve a variety of diseases And patients with clinical needs. Currently, children with neurogenic detrusor overactivity have very limited treatment options after failure of anticholinergic therapy and before surgery. If approved, BOTOX® will It is the first neurotoxin therapy approved for the treatment of children with neurogenic detrusor overactivity in response to anticholinergic drugs."
Paul F. Austin, MD, director of the Department of Pediatric Urology, Texas Children’s Hospital, and professor of urology at Baylor College of Medicine, said: "With time, many children with internal neurological diseases will develop bladder and kidney damage. It is very important. Current treatment options usually include anticholinergic drugs. In addition to surgery, long-term use needs to be carefully considered. BOTOX® treatment of pediatric patients with neurogenic detrusor overactivity shows promising clinical results. Will meet unmet and ongoing medical needs among children and adolescents."
Excessive neurogenic detrusor muscle activity results from the inability of the spinal cord and bladder to communicate effectively. As a result, the bladder muscles (detrusor muscles) contract involuntarily, increasing the pressure in the bladder and reducing the volume of the bladder, resulting in frequent and accidental leakage of urine. If clean intermittent catheterization and anticholinergic drugs are not adequately treated, this condition may require expanded cystoplasty (a wide-ranging surgical procedure to enlarge the bladder by using a patient’s intestine) to prevent Kidney damage.
There are many causes of excessive neurogenic detrusor muscle activity in children, such as transverse myelitis, spinal cord injury, and spina bifida. Among them, spina bifida is the most common cause, affecting 1,500 to 2,000 of the more than 4 million babies in the United States each year. More than 90% of patients with spina bifida have urinary symptoms.
Botox is developed by Aierjian (acquired by AbbVie). The main component of the drug is highly purified botulinum toxin type A, which is a nerve conduction blocker used to treat excessive Active muscles. Botox was first approved in 1989 for the treatment of face spasm and strabismus. It was approved for cervical dystonia in 2000. Later, it was further expanded into the field of beauty, including wrinkle reduction, face thinning, elimination of eyebrow lines and crow's feet. In recent years, Botox has also been approved to treat various indications such as upper limb spasm, chronic migraine, neurological incontinence, overactive bladder, spasm, and severe underarm sweating (underarm hyperhidrosis).
In the US, as of now, Botox has been approved by the FDA for 11 treatment indications. Over the past 30 years, more than 100 million bottles of Botox® and Botox® Cosmetic (Botulinum Toxin A) have been sold worldwide, and more than 3700 articles have been published in scientific and medical journals. Botox® neurotoxin is one of the most widely studied drugs in the world.