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FDA Approves PTC Therapeutics' KEBILIDI, First Gene Therapy Delivered Directly to the Brain for AADC Deficiency

• The FDA has granted accelerated approval to PTC Therapeutics' KEBILIDI, a gene therapy for AADC deficiency, marking the first such therapy delivered directly to the brain. • KEBILIDI works by using an adeno-associated virus to deliver a functional DOPA decarboxylase gene, restoring dopamine production in patients with AADC deficiency. • Clinical trials demonstrated promising results, with patients experiencing de novo dopamine synthesis and achieving motor development milestones following KEBILIDI treatment. • PTC Therapeutics received a rare disease priority review voucher with the approval and is preparing for the US launch, having identified centers of excellence and trained surgeons.

The U.S. Food and Drug Administration (FDA) has granted accelerated approval to KEBILIDI (eladocagene exuparvovec), a gene therapy developed by PTC Therapeutics, for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency in both children and adults. This marks a significant milestone as the first gene therapy approved in the US that is directly administered to the brain.
AADC deficiency is a rare genetic disorder that impairs motor function by disrupting dopamine synthesis, a critical neurotransmitter. KEBILIDI addresses this deficiency by utilizing a recombinant adeno-associated virus serotype 2 (AAV2) to deliver a functional DOPA decarboxylase (DDC) gene directly into the putamen of the brain. This increases AADC enzyme levels, restoring the brain's ability to produce dopamine.
The FDA's accelerated approval is based on data from the ongoing PTC-AADC-GT-002 global clinical trial, which demonstrated the safety and clinical efficacy of KEBILIDI. Patients treated with KEBILIDI showed evidence of de novo dopamine synthesis and achieved motor development milestones that are typically absent in individuals with AADC deficiency.
"PTC has once again pioneered a new approach to treating highly morbid neurologic diseases," said Matthew Klein, CEO of PTC Therapeutics. "I am proud of our team’s unwavering commitment to achieve this important regulatory milestone. We look forward to bringing this transformational gene therapy to children and adults with AADC deficiency in the US."
PTC Therapeutics is actively preparing for the launch of KEBILIDI in the US, including identifying centers of excellence and training surgeons on the precise neurosurgical procedure required for administration. With the biologics license application approval, the company has also been awarded a rare disease priority review voucher, which it intends to monetize.
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Reference News

[1]
FDA grants accelerated approval for PTC's AADC deficiency gene therapy
pharmaceutical-technology.com · Nov 14, 2024

FDA grants accelerated approval for PTC Therapeutics' KEBILIDI, the first US gene therapy directly delivered to the brai...

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