PTC Therapeutics Reports the US FDA’s Accelerated Approval of its Gene Therapy for AADC Deficiency
Shots:
- PTC Therapeutics’ brain-administered gene therapy has received the US FDA’s accelerated approval for AADC deficiency. The company also received a Rare Disease Priority Review Voucher, which it will monetize
- Approval was based on data from the ongoing PTC-AADC-GT-002 trial, with a confirmatory long-term follow-up planned. It will be commercialized across the US for children & adults under the brand name, Kebilidi (eladocagene exuparvovec-tneq), with preparations underway
- Kebilidi is a rAAV2-based gene therapy developed to transfer a functioning DDC gene to the putamen, boosting AADC enzyme levels and restoring dopamine production
Ref: PTC Therapeutics | Image: PTC Therapeutics
Related News:- PTC Therapeutics’ Upstaza (eladocagene exuparvovec) Receives NICE Recommendation for the Treatment of AADC Deficiency
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Disha is a content writer at PharmaShots. She is passionate and curious about recent updates and developments in MedTech and Pharma industry. She covers news related to clinical trial results and updates. She can be contacted at connect@pharmashots.com.
