PTC Therapeutics announced on November 13, 2024, that the U.S. Food and Drug Administration (FDA) granted accelerated approval for its gene therapy, KEBILIDI (eladocagene exuparvovec-tneq). This therapy is indicated for the treatment of children and adults with aromatic L-amino acid decarboxylase (AADC) deficiency, marking a pioneering achievement as the first gene therapy approved in the U.S. that is directly administered to the brain.
The approval comes with a broad label, covering the full spectrum of disease severity in both pediatric and adult patients. Clinical trial results demonstrated that following KEBILIDI administration, de novo synthesis of dopamine occurred, leading to the progressive acquisition of motor development milestones.
Alongside the Biologics License Application approval, PTC Therapeutics was granted a Rare Disease Priority Review Voucher (PRV). This PRV is a valuable asset that the company plans to monetize, providing additional financial flexibility.
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