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Kebilidi

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health1 year ago•2 min saved

FDA Approves PTC's Groundbreaking Brain-Delivered Gene Therapy for Ultrarare Disease

PTC Therapeutics has received FDA approval for Kebilidi, the first U.S. gene therapy administered directly in the brain, to treat aromatic L-amino acid decarboxylase (AADC) deficiency, a rare and fatal genetic disorder. Kebilidi delivers a functioning DDC gene to the brain's putamen to restore dopamine production, showing promising results in motor development. The approval is seen as a significant advancement in gene therapy, with PTC preparing for its U.S. launch and expecting substantial revenue. The company also received a rare disease priority review voucher from the FDA.

via FiercePharma|

#aadc-deficiency #fda #gene-therapy

health1 year ago•3 min saved

FDA Greenlights PTC's Groundbreaking Brain-Delivered Gene Therapy

The FDA has approved Kebilidi, the first gene therapy for treating aromatic L-amino acid decarboxylase (AADC) deficiency, a rare genetic disorder affecting neurotransmitter production. Administered via brain infusions, Kebilidi has shown efficacy in improving motor function in patients with severe AADC deficiency. The approval was granted under the Accelerated Approval pathway, with ongoing trials to confirm its clinical benefits. The therapy is developed by PTC Therapeutics, Inc., and uses the SmartFlow Neuro Cannula for administration.

via FDA.gov|

#aadc-deficiency #biotechnology #fda