FDA Approves Kebilidi, the first gene therapy indicated for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency.

The U.S. Food and Drug Administration has approved PTC Therapeutics' one-time gene therapy, called Kebilidi, for people with AADC deficiency.

FDA Approves Kebilidi, the first gene therapy indicated for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency.

This JAMA Insights discusses the US Food and Drug Administration approval of Kebilidi, eladocagene exuparvovec, for the treatment of aromatic L-amino acid decarboxylase deficiency in adult and pediatric patients.

FDA Approves Kebilidi for Aromatic L-Amino Acid Decarboxylase Deficiency  Physician's Weekly

Editor's note: This report was updated Aug. 25 to include PTC Therapeutics’ Kebilidi after a spokesperson disclosed the price of the gene therapy. With more and more expensive gene therapies hitting the market, one-dose, curative treatments are continuously shuffling the list of the U.S.’ highest-priced drugs.   | Multimillion-dollar one-dose therapies from Kyowa Kirin, CSL and Sarepta are just some of 2025's most expensive drugs in the U.S.

A drug approval this week illustrates the willingness of FDA’s Center for Biologics Evaluation and Research to lean into accelerated approvals of ge...

FDA Approves Kebilidi, the first gene therapy indicated for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency.

Recent FDA approvals of gene therapies like Beqvez and Kebilidi have sparked debates about million-dollar pricing and equitable access. Payers implement restrictive policies while innovative payment m...

Over 100 gene therapy clinical programs currently target neurological and neurodegenerative diseases, including Parkinson’s and Huntington’s. For adeno-associated virus (AAV)-based programs targeting the CNS, delivery remains a principal bottleneck. Direct intraparenchymal (IP) injection has proven effective for indications with highly focal pathology (Kebilidi/Upstaza for aromatic L-amino acid decarboxylase [AADC] deficiency) but requires invasive neurosurgery and achieves limited volumetric coverage of the target structure.

(1, 14) KEBILIDI is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of adult and pediatric patients with aromatic L amino acid decarboxylase (AADC) deficiency. ...

ClearPoint Neuro (NASDAQ:CLPT) has achieved significant milestones in its Biologics and Drug Delivery Portfolio. The company announced the first commercial gene therapy treatments in the US using their SmartFlow® Neuro Cannula for KEBILIDI™, a therapy for AADC deficiency. These groundbreaking procedures were performed at hospitals in Texas and Boston.Key achievements include: 17 global patients treated across 11 drug platforms in July, two additional partners accepted for FDA expedited review (bringing total to nine), and new ICD-10 codes creation for neuro infusion procedures effective October 1, 2025. The company currently supports over 60 active biopharma partners in clinical trials and preclinical studies.

Texas hospital uses Kebilidi gene therapy to treat girl, 3, with...  AADC News

Girl with AADC deficiency thriving after Kebilidi treatment  AADC News

Kebilidi linked to clinically meaningful motor gains: Analyses  AADC News

FDA approves Kebilidi for aromatic L-amino acid decarboxylase deficiency  Medical Xpress