ARTICLE | Regulation
Biotech expects to sell voucher after winning gene therapy’s approval for AADC deficiency based on 13-patient study
By Paul Bonanos, Director of Biopharma Intelligence
November 15, 2024 9:26 PM UTC
A drug approval this week illustrates the willingness of FDA’s Center for Biologics Evaluation and Research to lean into accelerated approvals of gene therapies for ultra-rare conditions, as the organization authorized use of a treatment from PTC based on data from a 13-patient study.
Late Wednesday, FDA granted accelerated approval to Kebilidi eladocagene exuparvovec to treat aromatic L-amino acid decarboxylase (AADC) deficiency, making it the first gene therapy approved by the agency for that disorder…
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- Source: https://www.biocentury.com/article/654225/ptc-wins-gene-therapy-approval-based-on-small-open-label-trial