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FDA Grants Accelerated Approval to PTC Therapeutics' KEBILIDI™, the First Gene Therapy for AADC Deficiency in the U.S.

NextEdge Admin

14 Nov 2024

2 min 42 sec

PTC Therapeutics, has received U.S. FDA accelerated approval for KEBILIDI™ (eladocagene exuparvovec-tneq), the first gene therapy in the United States directly administered to the brain for the treatment of AADC deficiency. This rare and debilitating genetic disorder prevents the synthesis of dopamine, a neurotransmitter essential for motor function, leading to severe physical, mental, and behavioral impairments.

KEBILIDI is designed to correct this underlying defect by delivering a functional DDC gene directly into the putamen, a part of the brain crucial for motor skills. Through a minimally invasive stereotactic surgical procedure, the therapy promotes the production of the AADC enzyme and the synthesis of dopamine, which has been shown to help patients progressively achieve critical motor development milestones.

AADC deficiency is a life-limiting condition that manifests early in life, often causing distressing symptoms such as oculogyric crises—episodes involving an involuntary upward gaze, muscle spasms, and inconsolability. The disorder severely impacts the quality of life, necessitating extensive support, including physical, occupational, and speech therapies, and sometimes surgical interventions for feeding and breathing challenges.

Matthew B. Klein, M.D., CEO of PTC Therapeutics, emphasized the significance of this approval, describing it as a “transformational therapy” for children and adults with AADC deficiency. Launch preparations are underway, with trained neurosurgeons at specialized centers ready to administer the therapy using the SmartFlow Neuro Cannula.

The FDA granted accelerated approval based on safety and efficacy results from PTC’s global clinical trial (PTC-AADC-GT-002), with ongoing long-term follow-up to confirm the therapy’s benefits. Additionally, PTC received a Rare Disease Priority Review Voucher, which it intends to monetize. This breakthrough represents a significant advancement in treating severe neurologic diseases.