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The Food and Drug Administration (FDA) has approved a new indication for onabotulinumtoxinA (Botox; Allergan, Madison, NJ)—treatment of upper limb spasticity in children, age 2 to 17 years. The decision came after a 6-month priority review, which istypically granted to therapies with potential to provide significant improvements in safety and effectiveness compared with current standard of care.
Approval is based on data from two phase 3 studies (NCT01603602, NCT01603615) a 12-week double-blind placebo controlled safety and efficacy study and a 1-year open-label extension study. Results of the 12-week study showed that onabotulinumtoxinA treatment (3 U/kg) resulted in significantly more improvement in spasticity vs placebo (-1.92 vs -1.21; P < .001) as measured by mean change in Modified Ashworth Scale-Bohannon (MAS-B) scores, on which decreases signify improvement of symptoms.
The approved recommended dose per treatment session is 3 U/kg to 6 U/kg divided among affected muscles of the upper limb, not to exceed the lower of 8 U/kg or 300 total units in a 3-month period. As noted on the drug label, treatment with onabotulinumA toxin does not replace physical or other rehabilitative therapy.
"Watching a child suffering with any degree of upper limb spasticity is very difficult," said David Nicholson, Chief Research and Development Officer, Allergan. "This FDA approval is special to all of us at Allergan because we can now provide children and their caregivers an advancement in pediatric care with Botox. This milestone underscores our constant focus on innovation and builds on our 30-years of research and development efforts with Botox since FDA approval of blepharospasm and strabismus in 1989. We also look forward to the FDA's decision on pediatric lower limb spasticity and continuing to serve our patients globally."
"Pediatric upper limb spasticity is a significant concern and can negatively impact a child's development and quality of life," said Mark Gormley, Jr. MD, Pediatric Rehabilitation Medicine Specialist, Gillette Children's Specialty Healthcare-St. Paul. "Because spasticity is particularly debilitating to growing children, it requires ongoing care. Botox has a well-established safety and efficacy profile, and I believe it will be an important treatment option in helping successfully manage upper limb spasticity in children and adolescents."
Stanley Jones P. Iyadurai, MD, PhD, FAAN
David S. Saperstein, MD
Arnold M. Salazar; Amanda M. Leisgang; Andrew A. Ortiz; and Jefferson W. Kinney, PhD