FDA Approves Expanded OnabotulinumtoxinA Label for Pediatric Spasticity

The Food and Drug Administration (FDA) approved a supplemental biologics license application (sBLA) with an expanded indication for onabotulinumtoxinA (Botox; Allergan, Chicago, IL), the treatment of spasticity in children.OnabotulinumtoxinA is a prescription medicine that is injected into muscles and used:
•    To treat increased muscle stiffness in individuals 2 years of age and more with spasticity 
•    To treat the abnormal head position and neck pain that happens with cervical dystonia (CD) in individuals 16 years and more 
•    To treat certain types of eye muscle problems (strabismus) or abnormal spasm of the eyelids (blepharospasm) in individuals 12 years and more

This label expansion is based on manufacturers selectively waiving orphan exclusivity marketing rights each company held for the use of their respective neurotoxins in the treatment of children with spasticity caused by cerebral palsy. OnabotulinumtoxinA was first approved in June 2019 for the treatment of pediatric patients with upper limb spasticity and in October 2019 for the treatment of pediatric patients with lower limb spasticity, excluding spasticity caused by cerebral palsy. OnabotulinumtoxinA has not been shown to improve upper extremity functional abilities, or range of motion at a joint affected by a fixed contracture. 

"Cerebral palsy is the most common cause of pediatric spasticity, which can have a profound impact on a child's development and quality of life. With its established safety and efficacy profile, we are pleased that Botox can now more broadly support physicians treating pediatric spasticity," said Mitchell F. Brin, MD, senior vice president, chief scientific officer, onabotulinumtoxinA & Neurotoxins, AbbVie. "Building upon our 30 years of research and development efforts with Botox, our commitment to neurotoxin innovation continues, and it is particularly rewarding to bring forth new treatments to advance care for pediatric patients." 

The safety and efficacy of onabotulinumtoxinA as treatment for lower limb spasticity for pediatric patients is supported by a phase 3 study with more than 300 participants, ages 2 to 17 years, with lower limb spasticity because of cerebral palsy. These trials included a 12-week, double-blind study and a 1-year open-label extension study.