FDA Approves PTC's Groundbreaking Brain-Delivered Gene Therapy for Ultrarare Disease
TL;DR Summary
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.
- PTC wins FDA approval for first brain-delivered gene therapy Kebilidi FiercePharma
- FDA approves PTC’s ultra-rare disease gene therapy for broader patient population than Europe Endpoints News
- US FDA approves PTC Therapeutics' gene therapy for ultra-rare disorder Reuters
- ClearPoint Neuro Announces FDA De Novo Marketing Authorization of SmartFlow Cannula for Direct Delivery of Gene Therapy to the Brain AccessWire
- PTC Wins First FDA Nod for Direct-to-Brain Gene Therapy, Targets Ultrarare Disease BioSpace
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