The US Food and Drug Administration (FDA) has granted accelerated approval for PTC Therapeutics’ KEBILIDI, the first gene therapy in the US that is directly delivered to the brain.
This therapy is designed to address the needs of children and adults with aromatic L-amino acid decarboxylase (AADC) deficiency.
The FDA’s accelerated approval is based on the safety and clinical efficacy findings from the ongoing PTC-AADC-GT-002 global clinical trial.
KEBILIDI works by replacing the defective gene and is delivered through a precise neurosurgical procedure to the putamen of the brain.
Clinical trials have shown promising results, with patients experiencing the de novo synthesis of dopamine and subsequent motor development milestones.
As part of the biologics license application approval, the company has also been awarded a rare disease priority review voucher, which it intends to monetise.
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KEBILIDI utilises a recombinant adeno-associated virus serotype 2 to deliver a functional DOPA decarboxylase gene directly into the brain, thereby increasing AADC enzyme levels and restoring dopamine production.
Preparations for the launch of KEBILIDI are ongoing, with centres of excellence identified and surgeons trained to administer the therapy.
AADC deficiency is a genetic disorder that severely impairs motor function by preventing dopamine synthesis, an essential neurotransmitter.
PTC Therapeutics CEO Matthew Klein said: “PTC has once again pioneered a new approach to treating highly morbid neurologic diseases.
“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.”
In October 2022, PTC Therapeutics signed a strategic financing partnership with Blackstone, valued at up to $1bn, to support the development of its product pipeline.
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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