Approval represents first gene therapy for treatment of AADC deficiency
By Lori Solomon HealthDay Reporter
TUESDAY, Nov. 26, 2024 (HealthDay News) — The U.S. Food and Drug Administration has approved Kebilidi (eladocagene exuparvovec-tneq) for the treatment of adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency.
The adeno-associated virus vector-based gene therapy is the first FDA-approved gene therapy for treatment of AADC deficiency, a rare genetic disorder affecting the production of some neurotransmitters. Kebilidi needs to be administered in a medical center that specializes in pediatric stereotactic neurosurgery. In one surgical session, four infusions are delivered to the brain region involved in motor control.
The accelerated approval was based on an open-label, single-arm clinical study that included 13 pediatric patients with a confirmed diagnosis of AADC deficiency. At baseline, all patients had no gross motor function (the most severe presentation of AADC deficiency) and decreased AADC activity in the plasma. Compared with untreated patients (natural history), eight of 12 patients treated with Kebilidi exhibited improvement in gross motor function at week 48.
“Clinical advancements in the field of gene therapy continue to lead to the discovery and availability of innovative treatment options for rare diseases that are otherwise difficult to manage,” Peter Marks, M.D., Ph.D., from the FDA Center for Biologics Evaluation and Research, said in a statement.
Approval of Kebilidi was granted to PTC Therapeutics.
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