The therapy, Kebilidi, was approved to treat adults and children with aromatic L-amino acid decarboxylase deficiency, a rare genetic disorder that affects the body’s ability to produce neurotransmitters that support motor function.
Kebilidi is administered via four infusions into a part of the brain involved in motor control. The one-time procedure must be performed at a medical center specializing in pediatric stereotactic neurosurgery, the FDA said.
The agency also authorized the use of the SmartFlow Neuro Cannula to administer the therapy.
PTC Therapeutics, the gene therapy’s maker, said it’s working with surgeons and centers of excellence in preparation for the therapy’s launch.
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