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# FDA Approves PTC Therapeutics’ Kebilidi
- URL: https://www.fdaweb.com/fda-approves-ptc-therapeutics-kebilidi/
- Published: 2024-11-14T12:00:00.000Z
- Updated: 2026-09-14T14:47:04.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5158186

FDA has approved PTC Therapeutics’ Kebilidi (eladocagene exuparvovec-tneq), an adeno-associated virus vector-based gene therapy indicated to treat adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency. An agency [statement](https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treatment-aromatic-l-amino-acid-decarboxylase-deficiency?ref=fdaweb.com) says Kebilidi is the first FDA-approved gene therapy to treat AADC deficiency.

The therapy’s safety and effectiveness were demonstrated in a clinical study involving 13 pediatric patients with a confirmed diagnosis of AADC deficiency. The most common adverse reactions are dyskinesia; fever; low blood pressure; anemia; increased saliva production; insomnia; low levels of potassium, phosphate, and/or magnesium; and procedural complications such as respiratory and cardiac arrest, FDA says.

Kebilidi was approved via the accelerated approval pathway and was granted priority review, orphan drug designation, and a rare pediatric disease priority review voucher by FDA.